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Spinal Muscular Atrophies of Childhood (Werdnig Hoffman Disease)

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)
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, Juvenile; Muscular Atrophy, Spinal, Infantile Chronic Form; Muscular Atrophy, Spinal, Intermediate Type; Muscular Atrophy, Spinal, Type I; Muscular Atrophy, Spinal, Type II; Muscular Atrophy, Spinal, Type III; Proximal Hereditary Motor Neuropathy Type I; SMA, Infantile Acute Form; Spinal Muscular Atrophy 1; Spinal Muscular Atrophy Type 2; Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III; Spinal Muscular Atrophy, Mild Childhood and Adolescent Form; 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; Infantile Muscular Atrophy; Juvenile Muscular Atrophy; Kugelberg Welander Disease; Kugelberg Welander Syndrome; Werdnig Hoffmann Disease; Juvenile Spinal Muscular Atrophy; Muscular Atrophy, Spinal, Infantile; Spinal Muscular Atrophy, Infantile; Spinal Muscular Atrophy, Juvenile
Networked: 190 relevant articles (8 outcomes, 20 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Sialorrhea
2. Pulmonary Atelectasis
3. Spinal Muscular Atrophies of Childhood (Werdnig Hoffman Disease)
4. Spinal Muscular Atrophy (Progressive Muscular Atrophy)
5. Neurodegenerative Diseases (Neurodegenerative Disease)

Experts

1. Messina, Sonia: 5 articles (01/2021 - 07/2008)
2. Swoboda, Kathryn J: 4 articles (01/2022 - 03/2014)
3. Bertini, Enrico: 4 articles (01/2021 - 07/2008)
4. D'Amico, Adele: 4 articles (01/2021 - 07/2008)
5. Mercuri, Eugenio: 4 articles (01/2021 - 07/2008)
6. Montes, Jacqueline: 4 articles (01/2021 - 10/2012)
7. Pane, Marika: 4 articles (01/2021 - 07/2008)
8. Iannaccone, Susan T: 4 articles (12/2019 - 01/2009)
9. van den Berg, Leonard H: 4 articles (12/2019 - 01/2009)
10. Bruno, Claudio: 3 articles (01/2021 - 01/2019)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Spinal Muscular Atrophies of Childhood:
1. nusinersenIBA
2. onabotulinum toxin AIBA
3. Guanidine (Guanidine Nitrate)FDA Link
4. Thyrotropin-Releasing Hormone (Protirelin)FDA Link
5. SugammadexIBA
6. Surface-Active Agents (Surfactants)IBA
7. Rocuronium (Zemuron)FDA LinkGeneric
8. Biomarkers (Surrogate Marker)IBA
9. Myosins (Myosin)IBA
10. DNA (Deoxyribonucleic Acid)IBA

Therapies and Procedures

1. Therapeutics
2. Injections
3. Noninvasive Ventilation
4. Perioperative Care
5. Resistance Training