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Spinal Muscular Atrophies of Childhood (Werdnig Hoffman Disease) Summary

Description: A group of recessively inherited diseases that feature progressive muscular atrophy and hypotonia. They are classified as type I (Werdnig-Hoffman disease), type II (intermediate form), and type III (Kugelberg-Welander disease). Type I is fatal in infancy, type II has a late infantile onset and is associated with survival into the second or third decade. Type III has its onset in childhood, and is slowly progressive. (J Med Genet 1996 Apr:33(4):281-3)

Also Known As: Werdnig Hoffman Disease; Kugelberg-Welander Disease; Spinal Muscular Atrophy Type I; Infantile Spinal Muscular Atrophy; Werdnig-Hoffmann Disease Show All >>

Networked: 130 relevant articles (3 outcomes, 12 trials/studies) for this Disease

Key Drugs and Agents for Spinal Muscular Atrophies of Childhood

Efficacy Chart >>
Drugs and Important Biological Agents (IBA) related to treatments:
  1. Guanidine (Guanidine Nitrate) : 1 outcome in 2 results : FDA 1
  2. Thyrotropin-Releasing Hormone (Protirelin) : 1 outcome in 2 results : FDA 2
  3. Sugammadex : 1 outcome in 2 results : IBA
  4. rocuronium (rocuronium bromide) : 1 outcome in 1 result : FDA 7 Generic
  5. Myosins (Myosin) : 2 studies in 3 results : IBA
  6. DNA (Deoxyribonucleic Acid) : 1 study in 7 results : IBA
  7. Arthrogryposis multiplex congenita : 1 study in 3 results : IBA
  8. Synaptophysin : 1 study in 2 results : IBA
  9. Nerve Growth Factor (NGF) : 1 study in 1 result : IBA
  10. Protein Isoforms (Isoforms) : 1 study in 1 result : IBA
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Diseases Related to Spinal Muscular Atrophies of Childhood

  1. Spinal Muscular Atrophies of Childhood (Werdnig Hoffman Disease)
  2. Muscular Dystrophies (Muscular Dystrophy)
  3. Spinal Muscular Atrophy (Progressive Muscular Atrophy)
  4. Muscular Diseases (Myopathy)
  5. Polymyositis
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Key Therapies for Spinal Muscular Atrophies of Childhood

Efficacy Chart >>
  1. Walkers : 1 study in 1 result
  2. Denervation : 4 results
  3. Tracheostomy : 3 results
  4. Gastrostomy : 3 results
  5. Cesarean Section (Caesarean Section) : 3 results
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