2. Disease Areas
  3. Musculoskeletal Disease
  4. Muscle Disease
  5. Neuromuscular Disease

Neuromuscular Disease

Neuromuscular disease is a heterogeneous group of disorders affecting the peripheral nervous system, neuromuscular junctions, muscles, or associated structures, resulting in muscle weakness, atrophy, and impaired motor and sensory function. These conditions arise from diverse etiologies including genetic, autoimmune, metabolic, and environmental factors, and can manifest as progressive muscle wasting, spasms, twitching, pain, numbness, and tingling. Examples include Charcot-Marie-Tooth disease, myasthenia gravis, Lambert-Eaton syndrome, botulism, amyotrophic lateral sclerosis, muscular dystrophy, and spinal muscular atrophy. While many neuromuscular diseases lack a cure, early diagnosis and management strategies can help alleviate symptoms, slow progression, and improve quality of life.

Neuromuscular Disease (1):

Cat. No. Product Name CAS No. Purity Chemical Structure
  • HY-145725A
    Baliforsen 1698048-23-5 98.12%
    Baliforsen (ISIS 5987690) is an antisense oligonucleotide (ASO) that inhibits DMPK mRNA. Baliforsen binds within exon 9 of the human DMPK transcript to promote RNase H1-mediated degradation Baliforsen can be used for the research of myotonic dystrophy type 1.
    Baliforsen