Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration and weakness due to mutations in the dystrophin gene, leading to the absence or dysfunction of the dystrophin protein essential for muscle cell stability. Primarily affecting males, with an incidence of approximately 1 in 3,500 male births, DMD typically manifests around age four with initial weakness in the pelvic and thigh muscles, rapidly progressing to involve the arms and other skeletal muscles. The disease results in significant functional decline, requiring assisted ventilation and ultimately leading to premature death. In addition to skeletal muscle involvement, DMD affects cardiac muscle, making it a multi-systemic condition. The lack of dystrophin renders muscle fibers more susceptible to damage, and over time, the body's ability to repair and regenerate muscle tissue is overwhelmed, resulting in muscle wasting and weakness. Despite ongoing research, no curative therapy is currently available.

References:

[1]. Duchenne Muscular Dystrophy. sciencedirect.

Duchenne Muscular Dystrophy (13):

Targets:	DNA/RNA Synthesis (5) Fluorescent Dye (4) Antibody-Oligonucleotide Conjugates (AOCs) (3) Transferrin Receptor (2) Dystrophin (2) Transmembrane Glycoprotein (1)

Cat. No.	Product Name	CAS No.	Purity	Chemical Structure

HY-P990051

Delpacibart	2653183-66-3	98.06%
Delpacibart is a humanized IgG1κ monoclonal antibody targeting the transferrin receptor TFRC. Delpacibart can be conjugated with the phosphorodiamidate morpholino oligonucleotide (PMO) Zotadirsen (HY-177972), which targets exon 44 of the dystrophin gene, to synthesize the antibody-oligonucleotide conjugate (AOC) Delpacibart zotadirsen (HY-177564). Delpacibart is suitable for use in Duchenne muscular dystrophy (DMD44) research.

HY-145724

Drisapersen	1251830-50-8
Drisapersen (Kyndrisa) is a 2^'-O-methyl phosphorothioate RNA antisense oligonucleotide that induces exon 51 skipping. Drisapersen induces skipping of exon 51 during Dystrophin pre-mRNA splicing, allowing the synthesis of partially functional Dystrophin. Drisapersen can be used in research related to Duchenne muscular dystrophy.

HY-177564

Delpacibart zotadirsen	2929240-20-8
Delpacibart zotadirsen (Del-zota), an antibody oligonucleotide conjugate (AOC), consists of a monoclonal antibody (Delpacibart) (HY-P990051) that binds to the transferrin receptor 1 (TfR1) conjugated to a phosphorodiamidate morpholino conjugate (PMO), Delpacibart zotadirsen is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Delpacibart zotadirsen is used for the study of myotonic dystrophy type 1 (DM1).

HY-177972

Zotadirsen
Zotadirsen, one of the components of the AOC Delpacibart zotadirsen (HY-177564), is composed of a phosphorodiamidate morpholino oligomers (PMOs) and SMCC linker (HY-42360). Zotadirsen specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Zotadirsen can be used to synthesize AOC drugs. Zotadirsen can also be used in the research of Duchenne muscular dystrophy (DM).

HY-177972B

FAM labled Zotadirsen
FAM labled Zotadirsenis a FAM labled Zotadirsen.

HY-177972C

Cy3 labled Zotadirsen
Cy3 labled Zotadirsen is a Cy3 labled Zotadirsen.

HY-177972A

Zotadirsen scrambled negative control
Zotadirsen scrambled negative control is the sequence scrambled negative control of Zotadirsen.

HY-147253

Brogidirsen	2409088-11-3
Brogidirsen (NS 089; NCNP 02) is a a dual-targeting antisense oligonucleotide. Brogidirsen can induce dystrophin protein experession. Brogidirsen can be used for the research of Duchenne muscular dystrophy.

HY-147253C

Brogidirsen sodium scrambled negative control
Brogidirsen sodium scrambled negative control is the sequence scrambled negative control of Brogidirsen sodium.

HY-147253D

FAM labled Brogidirsen sodium
FAM labled Brogidirsen sodiumis a FAM labled Brogidirsen sodium.

HY-147253E

Cy3 labled Brogidirsen sodium
Cy3 labled Brogidirsen sodium is a Cy3 labled Brogidirsen sodium.

HY-147253A

Brogidirsen sodium
Brogidirsen (NS 089; NCNP 02) sodium is a a dual-targeting antisense oligonucleotide. Brogidirsen sodium can induce dystrophin protein experession. Brogidirsen sodium can be used for the research of Duchenne muscular dystrophy.

HY-P992223

Anti-DAG1/Dystroglycan Antibody (DAG-6F4)
Anti-DAG1/Dystroglycan Antibody (DAG-6F4) is a human monoclonal antibody against DAG1/Dystroglycan. Anti-DAG1/Dystroglycan Antibody (DAG-6F4) can bind specifically to the α and β subunits of the dystroglycan protein complex. Anti-DAG1/Dystroglycan Antibody (DAG-6F4) is applicable to research related to muscular dystrophy.

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