Spinal Muscular Atrophies of Childhood (Werdnig Hoffman Disease)

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; HMN (Hereditary Motor Neuropathy) Proximal Type I; Kugelberg-Welander Syndrome; Muscular Atrophy, Infantile; Muscular Atrophy, Spinal, Type I; Muscular Atrophy, Spinal, Type II; Muscular Atrophy, Spinal, Type III; Proximal Hereditary Motor Neuropathy Type I; Spinal Muscular Atrophy 1; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III; Spinal Muscular Atrophy, Type 3; Spinal Muscular Atrophy, Type I; Spinal Muscular Atrophy, Type II; Spinal Muscular Atrophy, Type III; Type I Spinal Muscular Atrophy; Type II Spinal Muscular Atrophy; Type III Spinal Muscular Atrophy; Atrophies, Infantile Muscular; Atrophy, Infantile Muscular; Hoffman Disease, Werdnig; Hoffman Diseases, Werdnig; Infantile Muscular Atrophies; Infantile Muscular Atrophy; Kugelberg Welander Disease; Kugelberg Welander Syndrome; Kugelberg-Welander Syndromes; Werdnig Hoffman Diseases; Werdnig Hoffmann Disease; Juvenile Spinal Muscular Atrophy; Muscular Atrophy, Spinal, Infantile; Spinal Muscular Atrophy, Infantile; Spinal Muscular Atrophy, Juvenile
Networked: 130 relevant articles (3 outcomes, 12 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

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


1. Davis, Rebecca Hurst: 2 articles (12/2015 - 03/2014)
2. Swoboda, Kathryn J: 2 articles (12/2015 - 03/2014)
3. Gitlin, Jonathan D: 2 articles (06/2015 - 01/2012)
4. Petris, Michael J: 2 articles (06/2015 - 01/2012)
5. Weisman, Gary A: 2 articles (06/2015 - 01/2012)
6. Tizzano, Eduardo F: 2 articles (01/2013 - 04/2003)
7. Kubota, M: 2 articles (08/2000 - 01/2000)
8. Sakakihara, Y: 2 articles (08/2000 - 01/2000)
9. Oka, A: 2 articles (08/2000 - 01/2000)
10. Miller, Elizabeth A: 1 article (12/2015)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Spinal Muscular Atrophies of Childhood:
1. Guanidine (Guanidine Nitrate)FDA Link
2. Thyrotropin-Releasing Hormone (Protirelin)FDA Link
3. SugammadexIBA
4. rocuronium (rocuronium bromide)FDA LinkGeneric
5. Myosins (Myosin)IBA
6. DNA (Deoxyribonucleic Acid)IBA
7. Arthrogryposis multiplex congenitaIBA
8. SynaptophysinIBA
9. Nerve Growth Factor (NGF)IBA
10. Protein Isoforms (Isoforms)IBA

Therapies and Procedures

1. Walkers
2. Denervation
3. Tracheostomy
4. Gastrostomy
5. Cesarean Section (Caesarean Section)