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Results for "

Cerebrospinal

" in MedChemExpress (MCE) Product Catalog:

By Targets:	Adrenergic Receptor (1) Akt (1) Amyloid-β (5) Androgen Receptor (1) Bacterial (3) Beta-secretase (1) Biochemical Assay Reagents (2) Dihydrofolate reductase (DHFR) (1) Dopamine Receptor (5) Dopamine Transporter (4) Drug Metabolite (1) Endogenous Metabolite (15) FGFR (1) GABA Receptor (1) Histamine Receptor (1) HSV (1) Interleukin Related (1) Isotope-Labeled Compounds (3) LDLR (1) Microtubule/Tubulin (1) Monoamine Oxidase (1) Monoamine Transporter (7) Neuropeptide FF Receptor (1) NF-κB (1) Opioid Receptor (1) Oxytocin Receptor (1) Peptide-Drug Conjugates (PDCs) (1) Prion Protein (1) Prolyl Endopeptidase (PREP) (1) Reactive Oxygen Species (ROS) (1) Reference Standards (4) SOD (3) Sodium Channel (1) Tau Protein (3) TNF Receptor (1) Xanthine Oxidase (1) γ-secretase (3)
By Research Areas:	Cancer (6) Cardiovascular Disease (9) Infection (1) Inflammation/Immunology (3) Metabolic Disease (5) Neurological Disease (38)
Oligonucleotides:	Antisense Oligonucleotides (3) Cholesterol (1)

Inhibitors & Agonists

Peptides

MCE Kits

Inhibitory Antibodies

Natural
Products

Isotope-Labeled Compounds

Oligonucleotides

Cat. No.	Product Name	Target	Research Areas	Chemical Structure

HY-132580

Tofersen 2 Publications Verification BIIB067; ISIS-SOD1Rx; ISIS 333611	SOD	Neurological Disease
Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis .

HY-B0590

Tetrabenazine 1 Publications Verification Ro 1-9569	Monoamine Transporter Dopamine Receptor	Neurological Disease
Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-113289

Brassicasterol 1 Publications Verification	Akt Androgen Receptor Bacterial Drug Metabolite HSV	Infection Cardiovascular Disease Neurological Disease Cancer
Brassicasterol is a metabolite of Ergosterol and has cardiovascular protective effects. Brassicasterol exerts anticancer effects in prostate cancer through dual targeting of AKT and androgen receptor signaling pathways. Brassicasterol inhibits HSV-1 (IC₅₀=1.2 μM) and Mycobacterium tuberculosis. Brassicasterol also inhibits sterol δ 24-reductase, slowing the progression of atherosclerosis. Brassicasterol is also a cerebrospinal fluid biomarker for Alzheimer's disease .

HY-B0590S

Tetrabenazine-d6 Ro 1-9569-d₆	Monoamine Transporter	Neurological Disease
Tetrabenazine-d₆ (Deutetrabenazine) is a deuterium-labled Tetrabenazine (HY-B0590). Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-P4073

ANG1005 GRN1005; Paclitaxel trevatide	Peptide-Drug Conjugates (PDCs) LDLR	Cancer
ANG1005 (Paclitaxel trevatide) is a brain-penetrating peptide-drug conjugate. ANG1005, a taxane derivative, consists of three paclitaxel (HY-B0015) molecules covalently linked to Angiopep-2, designed to cross the blood-brain and blood-cerebrospinal barriers and to penetrate malignant cells via low density lipoprotein receptor-related protein (LRP1) transport system .

HY-113358

6-keto Prostaglandin F1α 1 Publications Verification	Endogenous Metabolite	Inflammation/Immunology
6-keto Prostaglandin F1α is an endogenous metabolite present in Cerebrospinal_Fluid, Urine and Blood that can be used for the research of Meningitis, Rheumatoid Arthritis and Cardiopulmonary Resuscitation .

HY-132580A

Tofersen sodium 2 Publications Verification BIIB067 sodium; ISIS-SOD1Rx sodium; ISIS 333611 sodium	SOD	Neurological Disease
Tofersen (BIIB067) sodium is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen sodium mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen sodium downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen sodium can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis .

HY-W096638A

Glycerophosphocholine (S)-Glycerolphosphocholine	Endogenous Metabolite	Neurological Disease Metabolic Disease Cancer
Glycerophosphocholine ((S)-Glycerolphosphocholine) is an orally active metabolite that crosses the blood-brain barrier. Glycerophosphocholine acts as a precursor of acetylcholine, a specific marker for phosphatidylcholine breakdown, a major water-soluble storage form of choline, and an intermediate in phosphatidylcholine metabolism. Glycerophosphocholine is specifically elevated in the cerebrospinal fluid of Alzheimer's disease models, and it also promotes growth hormone secretion and fat oxidation. Glycerophosphocholine is also a potential biomarker for breast cancer .

HY-P1248

Neuropeptide FF NPFF	Neuropeptide FF Receptor	Cardiovascular Disease Neurological Disease
Neuropeptide FF (NPFF), an octapeptide belonging to the RF-amide family of peptides, is a NPFF1 and NPFF2 receptors agonist with K_i values of 2.82 nM and 0.21 nM, respectively. Neuropeptide FF induces abstinence syndrome, exerts antiopioid and analgesic effects, releases via calcium-dependent mechanisms from rat spinal cord, regulates memory, autonomic function, and neuroendocrine function, modulates pain and opioid antinociception, reduces food intake, stimulates water intake, alters cardiovascular parameters, and shows differential activity in hypothalamic paraventricular nucleus neurons. Neuropeptide FF is present in mammalian central nervous system and periphery, with NPFF-immunoreactivity increases in rat cerebrospinal fluid during opiate tolerance, and its NPFF gene and NPFF-R2 gene are up-regulated in rat spinal cord and dorsal root ganglia during peripheral inflammation. Neuropeptide FF can be used for the research of opioid tolerance, morphine-induced analgesia, abstinence syndrome, pain, hypertension, nociception, inflammatory pain, and neuropathic pain .

HY-P3281

FGL peptide	FGFR	Neurological Disease
FGL peptide is a fibroblast growth factor receptor (FGFR) modulator and blood-brain barrier-penetrant. FGL peptide activates NCAM-FGFR and FGFR1 signaling pathways. FGL peptide alters expression of apoptosis, signal transduction and metabolism regulator genes in traumatic brain injury contexts. FGL peptide can be used for the research of traumatic brain injury .

HY-113468

3-O-Methyl-DL-DOPA 3-Methoxytyrosine	Endogenous Metabolite	Neurological Disease
3-O-Methyl-DL-DOPA is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Epilepsy, Purine Nucleoside Phosphorylase Deficiency and Aromatic L Amino Acid Decarboxylase Deficiency .

HY-14174

MRK-560 4 Publications Verification	γ-secretase	Neurological Disease Cancer
MRK-560, a chemical probe, is an orally active, brain barrier-penetrated γ-Secretase inhibitor, reducing Aβ peptide in rat brain and cerebrospinal fluid. MRK-560 decreases mutant NOTCH1 processing by selectively inhibiting PSEN1. MRK-560 can be used in studies of Alzheimer's disease and T-cell acute lymphoblastic leukaemia (T-ALL) .

HY-12242

GBR 12935 dihydrochloride 1 Publications Verification	Dopamine Transporter	Neurological Disease
GBR 12935 dihydrochloride is a potent, and selective dopamine reuptake inhibitor, with the binding constant (K_d) of 1.08 nM in COS-7 cells. GBR 12935 dihydrochloride stimulates the locomotion activity in different mice strains but fails to induce stereotypy. Thus, GBR 12935 dihydrochloride also prevents the d-Fenfluramine-induced head-twitch response in mice .

HY-109183

Luvadaxistat TAK-831	Xanthine Oxidase	Others
Luvadaxistat (TAK-831) is an orally active, highly selective, potent D-amino acid oxidase (DAAO) inhibitor. Luvadaxistat inhibits oxidative deamination of D-serine via the human recombinant DAAO enzyme with an IC₅₀ of 14 nM. Luvadaxistat significantly increases D-serine levels in the rodent brain, plasma, and cerebrospinal fluid. Luvadaxistat has the potential for schizophrenia research .

HY-B0590A

Tetrabenazine Racemate Ro 1-9569 Racemate	Monoamine Transporter Dopamine Receptor	Neurological Disease
Tetrabenazine (Ro 1-9569) Racemate is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine Racemate binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine Racemate weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine Racemate can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-122381

Kyotorphin 1 Publications Verification	Bacterial Endogenous Metabolite	Neurological Disease
Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

HY-P9995

Posdinemab JNJ-63733657	Tau Protein	Neurological Disease
Posdinemab (JNJ-63733657) is a humanized IgG1/κ monoclonal antibody that selectively targets phosphorylated tau (pT217). Posdinemab specifically binds to the pT217+tau epitope rich in the proline domain, blocks tau protein aggregation and seed propagation, and promotes the clearance of extracellular tau species. Posdinemab reduces the levels of free and total p217+tau in cerebrospinal fluid, thereby inhibiting the pathological propagation of tau protein and the formation of neurofibrillary tangles. Posdinemab can be used for the study of progressive supranuclear palsy syndrome and Alzheimer's disease (AD), especially for prodromal or mild AD disease .

HY-113517

Normetanephrine 1 Publications Verification (±)-Normetanephrine; DL-Normetanephrine	Endogenous Metabolite	Cardiovascular Disease
Normetanephrine is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Hypertension .

HY-113046S

5-Methyltetrahydrofolic acid-13C5 5-Methyl THF-¹³C₅; 5-MTHF-¹³C,d₅	Endogenous Metabolite	Metabolic Disease
5-Methyltetrahydrofolic acid- ¹³C₅ is the ¹³C-labeled 5-Methyltetrahydrofolic acid. 5-Methyltetrahydrofolic acid (5-Methyl THF) is a biologically active form of folic acid. 5-Methyltetrahydrofolic acid is a methylated derivate of tetrahydrofolate. 5-Methyltetrahydrofolic acid is the predominant natural dietary folate and the principal form of folate in plasma and cerebrospinal fluid .

HY-125999

EPI-589	Reactive Oxygen Species (ROS)	Neurological Disease
EPI-589, a quinone derivative, is a safe and well tolerated oxidoreductase enzyme inhibitor and a free radical scavenger, with blood-brain barrier permeable and orally available. EPI-589 is a redox-active neuroprotectant that effectively delays the symptoms of motor neuron disease in wobbler mice. EPI-589 can be used in amyotrophic lateral sclerosis (ALS) research .

HY-P1378A

β-Amyloid (1-43)(human) TFA	Amyloid-β	Neurological Disease
β-Amyloid (1-43)(human) TFA is more prone to aggregation and has higher toxic properties than the long-known Aβ1-42. β-Amyloid (1-43)(human) TFA shows a correlation with both sAPPα and sAPPβ. β-Amyloid (1-43)(human) TFA could be considered an added Alzheimer's disease (AD) biomarker together with the others already in use .

HY-14196

Toloxatone 1 Publications Verification MD 69276	Monoamine Oxidase	Neurological Disease
Toloxatone (MD 69276) is a reversible, selective MAO-A inhibitor that can cross the blood-brain barrier. Toloxatone increases the levels of serotonin (5-HT) and norepinephrine in the brain. Toloxatone reduces the immobility time in the forced swimming test in mice, inhibits killing behavior in rats without causing sedation, and shows a correlation between its free plasma concentration and cerebrospinal fluid concentration. Toloxatone is widely used in research related to depression, depressive disorders and Parkinson's disease .

HY-16632

ELND 006	γ-secretase Amyloid-β	Metabolic Disease
ELND 006 (Compound 30) is a metabolically stable γ-secretase inhibitor designed to selectively inhibit amyloid beta (Aβ) generation while sparing Notch signaling. It was developed through a synthetic strategy emphasizing diversity and chirality. ELND 006, along with its analog ELND007 (Compound 34), progressed into human clinical trials. In preclinical studies, both compounds demonstrated effective reduction of Aβ levels in vitro and in vivo. Comparisons with other γ-secretase inhibitors like Semagacestat, Begacestat, and Avagacestat underscored their potency and specificity in lowering Aβ levels in cerebrospinal fluid (CSF) of healthy human volunteers, suggesting potential therapeutic efficacy in Alzheimer's disease .

HY-29347

Ocrylate	Biochemical Assay Reagents	Cardiovascular Disease
Ocrylate is a tissue adhesive that contains an 8-carbon alkyl cyanoacrylate in its structure. Ocrylate can be used in research on gastric fundal varices, bone and cartilage grafting, cerebrospinal fluid leakage repair, and skin closure. Additionally, the rapid polymerization property of Ocrylate makes it suitable for inhibiting vascular embolization .

HY-113317

L-Xylulose	Endogenous Metabolite	Cardiovascular Disease
L-Xylulose is an endogenous metabolite present in Blood, Cerebrospinal_Fluid and Urine that can be used for the research of Ribose 5 Phosphate Isomerase Deficiency .

HY-W029659

1-Benzyl-1,2,3,4-tetrahydro-isoquinoline	Endogenous Metabolite	Neurological Disease
1-Benzyl-1,2,3,4-tetrahydro-isoquinoline is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Parkinson's Disease .

HY-113375

D-Ribofuranose 1 Publications Verification D-Ribose	Endogenous Metabolite	Cardiovascular Disease
D-Ribofuranose (D-Ribose) is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Ribose 5 Phosphate Isomerase Deficiency and Medium Chain Acyl Co A Dehydrogenase Deficiency .

HY-113468R

3-O-Methyl-DL-DOPA (Standard) 3-Methoxytyrosine (Standard)	Endogenous Metabolite Reference Standards	Neurological Disease
3-O-Methyl-DL-DOPA (Standard) is the analytical standard of 3-O-Methyl-DL-DOPA. This product is intended for research and analytical applications. 3-O-Methyl-DL-DOPA is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Epilepsy, Purine Nucleoside Phosphorylase Deficiency and Aromatic L Amino Acid Decarboxylase Deficiency [4].

HY-P5183

Hm1a	Sodium Channel	Neurological Disease
Hm1a is a venom peptide and a selective hNaV1.1 activator with an EC₅₀ of 7.5 nM. Hm1a enhances hNaV1.1 and hNaV1.3 channel currents via delayed inactivation. Hm1a restores action potential firing in Dravet syndrome GABAergic inhibitory interneurons, reduces interictal epileptiform discharges and whole-brain hyperexcitability, lowers seizure frequency, and rescues premature death in Dravet syndrome mice. Hm1a can be used for the research of neurological disease, such as Dravet syndrome .

HY-P1378

β-Amyloid (1-43)(human)	Amyloid-β	Neurological Disease
β-Amyloid (1-43)(human) is more prone to aggregation and has higher toxic properties than the long-known Aβ1-42. β-Amyloid (1-43)(human) shows a correlation with both sAPPα and sAPPβ. β-Amyloid (1-43)(human) could be considered an added Alzheimer's disease (AD) biomarker together with the others already in use .

HY-112636

Astrophloxine	Amyloid-β	Others
Astrophloxine is a fluorescent imaging probe capable of targeting antiparallel dimers. Astrophloxine can be used to detect aggregated Aβ in brain tissue and cerebrospinal fluid samples of Alzheimer's disease (AD) mice .

HY-117272A

(2R,3R)-2,3,4-Trihydroxybutanoic acid	Endogenous Metabolite	Others
(2R,3R)-2,3,4-Trihydroxybutanoic acid, also known as erythronic acid, is a naturally occurring organic acid found in the aqueous humor of the eye, as well as in urine, plasma, cerebrospinal fluid, and synovial fluid, prevalent in both healthy adults and children; it is produced through the oxidation of GlcNAc.

HY-132580S

Tofersen-d27 BIIB067-d₂₇; ISIS-SOD1Rx-d₂₇; ISIS 333611-d₂₇	Isotope-Labeled Compounds SOD	Neurological Disease
Tofersen-d₂₇ (BIIB067-d₂₇) is the deuterium labeled Tofersen (HY-132580). Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.

HY-W768333

D-Ribofuranose-13C5 D-Ribose-¹³C₅	Isotope-Labeled Compounds Endogenous Metabolite	Cardiovascular Disease
D-Ribofuranose- ¹³C₅ (D-Ribose- ¹³C₅) is the ¹³C-labeled D-Ribofuranose (HY-113375). D-Ribofuranose (D-Ribose) is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Ribose 5 Phosphate Isomerase Deficiency and Medium Chain Acyl Co A Dehydrogenase Deficiency .

HY-12242A

GBR 12935	Dopamine Transporter	Neurological Disease
GBR 12935 is a potent, and selective dopamine reuptake inhibitor, with the binding constant (K_d) of 1.08 nM in COS-7 cells. GBR 12935 stimulates the locomotion activity in different mice strains but fails to induce stereotypy. Thus, GBR 12935 also prevents the d-Fenfluramine-induced head-twitch response in mice .

HY-16525

XEN-2174	Monoamine Transporter Adrenergic Receptor	Neurological Disease
XEN-2174 is a noradrenaline transporter (NET) inhibitor. XEN-2174 inhibits the reuptake of noradrenaline through non-competitive inhibition, increasing the concentration of noradrenaline in the synaptic cleft, thereby activating α2-adrenergic receptor at the spinal level and exerting analgesic effects. XEN-2174 exhibits long-lasting analgesic effects in models of neuropathic pain and postoperative pain in rats. XEN-2174 can be used in pain research .

HY-B0590E

Tetrabenazine mesylate Ro 1-9569 mesylate	Monoamine Transporter Dopamine Receptor	Neurological Disease
Tetrabenazine (Ro 1-9569) mesylate is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine mesylate binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine mesylate weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine mesylate can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-B0590R

Tetrabenazine (Standard) Ro 1-9569 (Standard)	Reference Standards Monoamine Transporter Dopamine Receptor	Neurological Disease
Tetrabenazine (Standard) is the analytical standard of Tetrabenazine (HY-B0590). This product is intended for research and analytical applications. Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-113517R

Normetanephrine (Standard) (±)-Normetanephrine (Standard); DL-Normetanephrine (Standard)	Endogenous Metabolite Reference Standards	Cardiovascular Disease
Normetanephrine (Standard) is the analytical standard of Normetanephrine. This product is intended for research and analytical applications. Normetanephrine is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Hypertension .

HY-B0590S3

Tetrabenazine-d7 TBZ-d7-d₇; Ro 1-9569-d₇	Isotope-Labeled Compounds Monoamine Transporter Dopamine Receptor	Neurological Disease
Tetrabenazine-d₇ (TBZ-d₇-d₇) is deuterium labeled Tetrabenazine (HY-B0590). Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-165081

Docosaenoyl ethanolamide N-(2-Hydroxyethyl)-13Z-docosenamide	Biochemical Assay Reagents	Others
Docosaenoyl ethanolamide (N-(2-Hydroxyethyl)-13Z-docosenamide) is one of a fatty N-acyl ethanolamines. Ethanolamines can be detected at relatively high levels in rat cerebrospinal fluid .

HY-115358

Lignoceroyl Ethanolamide Tetracosanoic acid monoethanolamide	Endogenous Metabolite	Metabolic Disease
Lignoceroyl ethanolamide is a member of the family of fatty N-acyl ethanolamines collectively called endocannabinoids. Whereas lignoceric acid has been detected at relatively high levels in rat cerebrospinal fluid, the specific role and relative importance of its ethanolamine metabolite have not been determined.

HY-P10498

[Des-Tyr1]-gamma-Endorphin	Opioid Receptor	Neurological Disease
[Des-Tyr1]-gamma-Endorphin is a neuropeptide, which can be found in human cerebrospinal fluid. [Des-Tyr1]-gamma-Endorphin exhibits antidepressant efficacy, facilitates the extinction of active avoidance and attenuates the passive avoidance behavior in rats .

HY-122381R

Kyotorphin (Standard)	Bacterial Endogenous Metabolite Reference Standards	Neurological Disease
Kyotorphin (Standard) is the analytical standard of Kyotorphin. This product is intended for research and analytical applications. Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

HY-100740A

Lanabecestat hydrochloride AZD-3293 hydrochloride; LY3314814 hydrochloride	Beta-secretase	Neurological Disease
Lanabecestat (AZD3293; LY3314814) hydrochloride is a potent BACE1 inhibitor that has been investigated for its potential as a modifying treatment for Alzheimer's disease. Lanabecestat (hydrochloride) demonstrated significant dose- and time-dependent reductions in concentrations of amyloid beta peptides in plasma, cerebrospinal fluid, and brain. Lanabecestat (hydrochloride) was also shown to produce reductions in Aβ neuritic plaque burden without demonstrating clinical benefits or slowing the progression of Alzheimer's disease pathophysiology.

HY-12242B

GBR 12935 fumarate	Dopamine Transporter	Neurological Disease
GBR 12935 fumarate is a potent, and selective dopamine reuptake inhibitor, with the binding constant (K_d) of 1.08 nM in COS-7 cells. GBR 12935 fumarate stimulates the locomotion activity in different mice strains but fails to induce stereotypy. Thus, GBR 12935 fumarate also prevents the d-Fenfluramine-induced head-twitch response in mice .

HY-12242R

GBR 12935 dihydrochloride (Standard)	Dopamine Transporter	Neurological Disease
GBR 12935 (dihydrochloride) (Standard) is the analytical standard of GBR 12935 (dihydrochloride). This product is intended for research and analytical applications. GBR 12935 dihydrochloride is a potent, and selective dopamine reuptake inhibitor, with the binding constant (Kd) of 1.08 nM in COS-7 cells. GBR 12935 dihydrochloride stimulates the locomotion activity in different mice strains but fails to induce stereotypy. Thus, GBR 12935 dihydrochloride also prevents the d-Fenfluramine-induced head-twitch response in mice .

HY-117637

ALS-I-41	Oxytocin Receptor	Neurological Disease
ALS-I-41 is a novel, potent and selective oxytocin receptor antagonist with the potential to modulate biological activities related to social behavior and mental disorders. ALS-I-41 is being evaluated for behavioral pharmacology experiments in non-human primates and can be administered via intranasal or intramuscular injection. The central nervous system penetration and metabolic rate of ALS-I-41 were studied by mass spectrometry analysis in cerebrospinal fluid and plasma of macaque monkeys .

HY-120269

CAY10512	NF-κB TNF Receptor Interleukin Related	Neurological Disease Inflammation/Immunology
CAY10512 is a NF-κB inhibitor. CAY10512 can suppress the upregulation of NF-κB-sensitive proinflammatory miRNAs (miRNA-9, miRNA-125b, miRNA-146a, miRNA-155) in cerebrospinal fluid and extracellular fluid. CAY10512 significantly reduces the release of pro-inflammatory cytokines (such as TNF-α, MCP-1, IL-8, IL-6). CAY10512 can be used for researchs on neuroinflammation, islet transplantation and microRNA regulation .

HY-16633

ELND 007	γ-secretase Amyloid-β	Metabolic Disease
ELND 007 (Compound 34) is a metabolically stable γ-secretase inhibitor designed to selectively inhibit amyloid beta (Aβ) generation while minimizing Notch inhibition. Developed alongside ELND 006 (Compound 30), it underwent human clinical trials following a synthetic strategy emphasizing diversity and chirality. In preclinical studies, ELND 007 showed promising in vitro and in vivo characteristics, effectively reducing Aβ levels. Comparative studies with other γ-secretase inhibitors like Semagacestat, Begacestat, and Avagacestat highlighted its efficacy in lowering Aβ production, particularly demonstrated by reduced Aβ levels in the cerebrospinal fluid (CSF) of healthy human volunteers, suggesting potential therapeutic benefit for Alzheimer's disease .

Cat. No.	Product Name	Target	Research Area

HY-P4073

ANG1005 GRN1005; Paclitaxel trevatide	Peptide-Drug Conjugates (PDCs) LDLR	Cancer
ANG1005 (Paclitaxel trevatide) is a brain-penetrating peptide-drug conjugate. ANG1005, a taxane derivative, consists of three paclitaxel (HY-B0015) molecules covalently linked to Angiopep-2, designed to cross the blood-brain and blood-cerebrospinal barriers and to penetrate malignant cells via low density lipoprotein receptor-related protein (LRP1) transport system .

HY-P1248

Neuropeptide FF NPFF	Neuropeptide FF Receptor	Cardiovascular Disease Neurological Disease
Neuropeptide FF (NPFF), an octapeptide belonging to the RF-amide family of peptides, is a NPFF1 and NPFF2 receptors agonist with K_i values of 2.82 nM and 0.21 nM, respectively. Neuropeptide FF induces abstinence syndrome, exerts antiopioid and analgesic effects, releases via calcium-dependent mechanisms from rat spinal cord, regulates memory, autonomic function, and neuroendocrine function, modulates pain and opioid antinociception, reduces food intake, stimulates water intake, alters cardiovascular parameters, and shows differential activity in hypothalamic paraventricular nucleus neurons. Neuropeptide FF is present in mammalian central nervous system and periphery, with NPFF-immunoreactivity increases in rat cerebrospinal fluid during opiate tolerance, and its NPFF gene and NPFF-R2 gene are up-regulated in rat spinal cord and dorsal root ganglia during peripheral inflammation. Neuropeptide FF can be used for the research of opioid tolerance, morphine-induced analgesia, abstinence syndrome, pain, hypertension, nociception, inflammatory pain, and neuropathic pain .

HY-P3281

FGL peptide	FGFR	Neurological Disease
FGL peptide is a fibroblast growth factor receptor (FGFR) modulator and blood-brain barrier-penetrant. FGL peptide activates NCAM-FGFR and FGFR1 signaling pathways. FGL peptide alters expression of apoptosis, signal transduction and metabolism regulator genes in traumatic brain injury contexts. FGL peptide can be used for the research of traumatic brain injury .

HY-122381

Kyotorphin 1 Publications Verification	Bacterial Endogenous Metabolite	Neurological Disease
Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

HY-P1378A

β-Amyloid (1-43)(human) TFA	Amyloid-β	Neurological Disease
β-Amyloid (1-43)(human) TFA is more prone to aggregation and has higher toxic properties than the long-known Aβ1-42. β-Amyloid (1-43)(human) TFA shows a correlation with both sAPPα and sAPPβ. β-Amyloid (1-43)(human) TFA could be considered an added Alzheimer's disease (AD) biomarker together with the others already in use .

HY-P5183

Hm1a	Sodium Channel	Neurological Disease
Hm1a is a venom peptide and a selective hNaV1.1 activator with an EC₅₀ of 7.5 nM. Hm1a enhances hNaV1.1 and hNaV1.3 channel currents via delayed inactivation. Hm1a restores action potential firing in Dravet syndrome GABAergic inhibitory interneurons, reduces interictal epileptiform discharges and whole-brain hyperexcitability, lowers seizure frequency, and rescues premature death in Dravet syndrome mice. Hm1a can be used for the research of neurological disease, such as Dravet syndrome .

HY-P1378

β-Amyloid (1-43)(human)	Amyloid-β	Neurological Disease
β-Amyloid (1-43)(human) is more prone to aggregation and has higher toxic properties than the long-known Aβ1-42. β-Amyloid (1-43)(human) shows a correlation with both sAPPα and sAPPβ. β-Amyloid (1-43)(human) could be considered an added Alzheimer's disease (AD) biomarker together with the others already in use .

HY-16525

XEN-2174	Monoamine Transporter Adrenergic Receptor	Neurological Disease
XEN-2174 is a noradrenaline transporter (NET) inhibitor. XEN-2174 inhibits the reuptake of noradrenaline through non-competitive inhibition, increasing the concentration of noradrenaline in the synaptic cleft, thereby activating α2-adrenergic receptor at the spinal level and exerting analgesic effects. XEN-2174 exhibits long-lasting analgesic effects in models of neuropathic pain and postoperative pain in rats. XEN-2174 can be used in pain research .

HY-P10498

[Des-Tyr1]-gamma-Endorphin	Opioid Receptor	Neurological Disease
[Des-Tyr1]-gamma-Endorphin is a neuropeptide, which can be found in human cerebrospinal fluid. [Des-Tyr1]-gamma-Endorphin exhibits antidepressant efficacy, facilitates the extinction of active avoidance and attenuates the passive avoidance behavior in rats .

HY-122381R

Kyotorphin (Standard)	Bacterial Endogenous Metabolite Reference Standards	Neurological Disease
Kyotorphin (Standard) is the analytical standard of Kyotorphin. This product is intended for research and analytical applications. Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

HY-P1383

Rac1 Inhibitor F56, control peptide	Peptides	Others
Rac1 Inhibitor F56, control peptide is a peptide containing residues 45-60 of Rac1. Rac1 Inhibitor F56, control peptide contains a Trp ⁵⁶ to Phe ⁵⁶ mutation. Rac1 Inhibitor F56, control peptide has no effect on Rac1 interaction with its guanine nucleotide exchange factors (GEFs) .

Cat. No.	Product Name	Target	Research Area	Image

HY-P9995

Posdinemab JNJ-63733657	Tau Protein	Neurological Disease
Posdinemab (JNJ-63733657) is a humanized IgG1/κ monoclonal antibody that selectively targets phosphorylated tau (pT217). Posdinemab specifically binds to the pT217+tau epitope rich in the proline domain, blocks tau protein aggregation and seed propagation, and promotes the clearance of extracellular tau species. Posdinemab reduces the levels of free and total p217+tau in cerebrospinal fluid, thereby inhibiting the pathological propagation of tau protein and the formation of neurofibrillary tangles. Posdinemab can be used for the study of progressive supranuclear palsy syndrome and Alzheimer's disease (AD), especially for prodromal or mild AD disease .

HY-P991957

PRN100	Prion Protein	Neurological Disease
PRN100 is a fully humanized monoclonal antibody against cellular prion protein (PrPᶜ), belonging to the IgG_4κ isotype. By binding to and stabilizing cellular prion protein, PRN100 is designed to prevent cellular prion protein from being recruited and converted into pathogenic, self-propagating prion (PrPˢᶜ) aggregates. PRN100 can be used for the study of Creutzfeldt-Jakob disease.

HY-P99648A

Gosuranemab (powder) BMS-986168 (powder); IPN007 (powder); BIIB092 (powder)	Tau Protein	Neurological Disease
Gosuranemab (BMS-986168; IPN007; BIIB092) (powder) is a humanised IgG4 anti-tau monoclonal antibody. Gosuranemab (powder) neutralizes the extracellular tau protein, inhibiting the spread and aggregation of pathological tau protein. Gosuranemab (powder) can be used for the research of progressive supranuclear palsy and early Alzheimer’s disease .

HY-P990995A

Etalanetug (Mouse IgG2a) 7G6 Antibody; 7G6-IgG2a	Tau Protein	Neurological Disease
Etalanetug (Mouse IgG2a) (7G6 Antibody) is a mouse monoclonal antibody targeting the HVPGG motif in the microtubule-binding domain of tau protein. Etalanetug (Mouse IgG2a) reduces the levels of insoluble tau protein in multiple brain regions and inhibits the seeding and spread of pathological tau protein. Etalanetug (Mouse IgG2a) is applicable to research related to Alzheimer's disease .

Cat. No.	Product Name	Category	Target	Chemical Structure

HY-113289

Brassicasterol 1 Publications Verification	Infection Animals Classification of Application Fields Disease Research Fields Steroids Source Classification Cancer	Akt Androgen Receptor Bacterial Drug Metabolite HSV
Brassicasterol is a metabolite of Ergosterol and has cardiovascular protective effects. Brassicasterol exerts anticancer effects in prostate cancer through dual targeting of AKT and androgen receptor signaling pathways. Brassicasterol inhibits HSV-1 (IC₅₀=1.2 μM) and Mycobacterium tuberculosis. Brassicasterol also inhibits sterol δ 24-reductase, slowing the progression of atherosclerosis. Brassicasterol is also a cerebrospinal fluid biomarker for Alzheimer's disease .

HY-113358

6-keto Prostaglandin F1α 1 Publications Verification	Classification of Application Fields Ketones, Aldehydes, Acids Endogenous metabolite Inflammation/Immunology Disease Research Fields Source Classification	Endogenous Metabolite
6-keto Prostaglandin F1α is an endogenous metabolite present in Cerebrospinal_Fluid, Urine and Blood that can be used for the research of Meningitis, Rheumatoid Arthritis and Cardiopulmonary Resuscitation .

HY-W096638A

Glycerophosphocholine (S)-Glycerolphosphocholine	Structural Classification Human Gut Microbiota Metabolites Ketones, Aldehydes, Acids Endogenous metabolite Source Classification	Endogenous Metabolite
Glycerophosphocholine ((S)-Glycerolphosphocholine) is an orally active metabolite that crosses the blood-brain barrier. Glycerophosphocholine acts as a precursor of acetylcholine, a specific marker for phosphatidylcholine breakdown, a major water-soluble storage form of choline, and an intermediate in phosphatidylcholine metabolism. Glycerophosphocholine is specifically elevated in the cerebrospinal fluid of Alzheimer's disease models, and it also promotes growth hormone secretion and fat oxidation. Glycerophosphocholine is also a potential biomarker for breast cancer .

HY-113468

3-O-Methyl-DL-DOPA 3-Methoxytyrosine	Monophenols Neurological Disease Classification of Application Fields Phenols Endogenous metabolite Disease Research Fields Source Classification	Endogenous Metabolite
3-O-Methyl-DL-DOPA is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Epilepsy, Purine Nucleoside Phosphorylase Deficiency and Aromatic L Amino Acid Decarboxylase Deficiency .

HY-122381

Kyotorphin 1 Publications Verification	Monophenols Neurological Disease Classification of Application Fields Phenols Endogenous metabolite Disease Research Fields Source Classification	Bacterial Endogenous Metabolite
Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

HY-113517

Normetanephrine 1 Publications Verification (±)-Normetanephrine; DL-Normetanephrine	Cardiovascular Disease Monophenols Classification of Application Fields Phenols Endogenous metabolite Disease Research Fields Source Classification	Endogenous Metabolite
Normetanephrine is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Hypertension .

HY-113317

L-Xylulose	Cardiovascular Disease Human Gut Microbiota Metabolites Microorganisms Classification of Application Fields Endogenous metabolite Disease Research Fields Saccharides Monosaccharides Source Classification	Endogenous Metabolite
L-Xylulose is an endogenous metabolite present in Blood, Cerebrospinal_Fluid and Urine that can be used for the research of Ribose 5 Phosphate Isomerase Deficiency .

HY-W029659

1-Benzyl-1,2,3,4-tetrahydro-isoquinoline	Alkaloids Neurological Disease Classification of Application Fields Endogenous metabolite Isoquinoline Alkaloids Disease Research Fields Source Classification	Endogenous Metabolite
1-Benzyl-1,2,3,4-tetrahydro-isoquinoline is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Parkinson's Disease .

HY-113468R

3-O-Methyl-DL-DOPA (Standard) 3-Methoxytyrosine (Standard)	Structural Classification Monophenols Phenols Endogenous metabolite Source Classification	Endogenous Metabolite Reference Standards
3-O-Methyl-DL-DOPA (Standard) is the analytical standard of 3-O-Methyl-DL-DOPA. This product is intended for research and analytical applications. 3-O-Methyl-DL-DOPA is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Epilepsy, Purine Nucleoside Phosphorylase Deficiency and Aromatic L Amino Acid Decarboxylase Deficiency [4].

HY-113517R

Normetanephrine (Standard) (±)-Normetanephrine (Standard); DL-Normetanephrine (Standard)	Structural Classification Monophenols Phenols Endogenous metabolite Source Classification	Endogenous Metabolite Reference Standards
Normetanephrine (Standard) is the analytical standard of Normetanephrine. This product is intended for research and analytical applications. Normetanephrine is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Hypertension .

HY-122381R

Kyotorphin (Standard)	Structural Classification Monophenols Phenols Endogenous metabolite Source Classification	Bacterial Endogenous Metabolite Reference Standards
Kyotorphin (Standard) is the analytical standard of Kyotorphin. This product is intended for research and analytical applications. Kyotorphin is an endogenou neuroactive dipeptide with analgesic properties. Kyotorphin possesses anti-inflammatory and antimicrobial activity. Kyotorphin levels in cerebro-spinal fluid correlate negatively with the progression of neurodegeneration in Alzheimer's Disease patients .

Cat. No.	Product Name	Chemical Structure

HY-B0590S

Tetrabenazine-d6

Tetrabenazine-d₆ (Deutetrabenazine) is a deuterium-labled Tetrabenazine (HY-B0590). Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

HY-113046S

5-Methyltetrahydrofolic acid-13C5
5-Methyltetrahydrofolic acid- ¹³C₅ is the ¹³C-labeled 5-Methyltetrahydrofolic acid. 5-Methyltetrahydrofolic acid (5-Methyl THF) is a biologically active form of folic acid. 5-Methyltetrahydrofolic acid is a methylated derivate of tetrahydrofolate. 5-Methyltetrahydrofolic acid is the predominant natural dietary folate and the principal form of folate in plasma and cerebrospinal fluid .

HY-132580S

Tofersen-d27

Tofersen-d₂₇ (BIIB067-d₂₇) is the deuterium labeled Tofersen (HY-132580). Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.

HY-W768333

D-Ribofuranose-13C5
D-Ribofuranose- ¹³C₅ (D-Ribose- ¹³C₅) is the ¹³C-labeled D-Ribofuranose (HY-113375). D-Ribofuranose (D-Ribose) is an endogenous metabolite present in Cerebrospinal_Fluid that can be used for the research of Ribose 5 Phosphate Isomerase Deficiency and Medium Chain Acyl Co A Dehydrogenase Deficiency .

HY-B0590S3

Tetrabenazine-d7

Tetrabenazine-d₇ (TBZ-d₇-d₇) is deuterium labeled Tetrabenazine (HY-B0590). Tetrabenazine (Ro 1-9569) is a brain-penetrant and orally active VMAT2-selective ligand with human VMAT2 K_i 100 nM. Tetrabenazine binds VMAT2 to block monoamine uptake into synaptic vesicles, potentiates cytoplasmic monoamine degradation. Tetrabenazine weakly blocks dopamine D2 receptors, and increases dopamine turnover via elevated cerebrospinal fluid homovanillic acid. Tetrabenazine can be used for the research of Huntington’s disease, tardive dyskinesia, and Tourette’s syndrome .

Cat. No.	Product Name		Classification

HY-132580

Tofersen 2 Publications Verification BIIB067; ISIS-SOD1Rx; ISIS 333611		Antisense Oligonucleotides
Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis .

HY-113289

Brassicasterol 1 Publications Verification		Cholesterol
Brassicasterol is a metabolite of Ergosterol and has cardiovascular protective effects. Brassicasterol exerts anticancer effects in prostate cancer through dual targeting of AKT and androgen receptor signaling pathways. Brassicasterol inhibits HSV-1 (IC₅₀=1.2 μM) and Mycobacterium tuberculosis. Brassicasterol also inhibits sterol δ 24-reductase, slowing the progression of atherosclerosis. Brassicasterol is also a cerebrospinal fluid biomarker for Alzheimer's disease .

HY-132580A

Tofersen sodium 2 Publications Verification BIIB067 sodium; ISIS-SOD1Rx sodium; ISIS 333611 sodium		Antisense Oligonucleotides
Tofersen (BIIB067) sodium is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen sodium mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen sodium downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen sodium can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis .

HY-132580S

Tofersen-d27 BIIB067-d₂₇; ISIS-SOD1Rx-d₂₇; ISIS 333611-d₂₇		Antisense Oligonucleotides
Tofersen-d₂₇ (BIIB067-d₂₇) is the deuterium labeled Tofersen (HY-132580). Tofersen (BIIB067) is an antisense oligonucleotide and SOD1 mRNA inhibitor with an IC₅₀ of 320 pM. Tofersen mediates RNase H-dependent degradation of SOD1 mRNA to reduce SOD1 protein levels in cerebrospinal fluid and serum. Tofersen downregulates cerebrospinal fluid neurofilament light chain, neurofilament heavy chain, amyloid-beta 1-40, amyloid-beta 1-42, neuropeptide Y, ubiquitin C-terminal hydrolase L1, neuropentraxins 1, 2, R, corticotropin-releasing hormone, IL-15, and serum neurofilament light chain, neurofilament heavy chain. Tofersen can be used for the research of superoxide dismutase 1-associated amyotrophic lateral sclerosis.

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