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Spinal Muscular Atrophies of Childhood
A group of recessive 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)
Drugs by status
Approved for this indication (2)
Phase 3 trials (1)
Phase 2 trials (10)
Other trials (1)
Organizations
Organization Involved with Phase 4 Indications (1)
Organization Involved with Phase 3 Indications (1)
Organization Involved with Phase 2 Indications (15)
- Roche
- Leadiant Biosciences, Inc.
- Westat
- National Institutes of Health (NIH)
- National Institute of Neurological Disorders and Stroke (NINDS)
- Bambino Gesù Hospital and Research Institute
- Johns Hopkins University
- Stanford University
- University of Tehran
- Utrecht University
- University of Utah
- Isfahan University
- Families of Spinal Muscular Atrophy
- CHR Citadelle
- Scholar Rock, Inc.
Organization Involved with Phase 1 Indications (1)