Muscular Dystrophies
A heterogeneous group of inherited MYOPATHIES, characterized by wasting and weakness of the SKELETAL MUSCLE. They are categorized by the sites of MUSCLE WEAKNESS; AGE OF ONSET; and INHERITANCE PATTERNS.
Subtype terms (9)
More specific conditions that are subtypes of Muscular Dystrophies in the MeSH hierarchy.
- Distal Myopathies
- Glycogen Storage Disease Type VII
2 drugs (1 approved, 1 experimental)
- Muscular Dystrophies, Limb-Girdle
20 drugs (5 approved, 15 experimental)
- Muscular Dystrophy, Duchenne
113 drugs (46 approved, 67 experimental)
- Muscular Dystrophy, Emery-Dreifuss
- Muscular Dystrophy, Facioscapulohumeral
16 drugs (7 approved, 9 experimental)
- Muscular Dystrophy, Oculopharyngeal
4 drugs (1 approved, 3 experimental)
- Myotonic Dystrophy
18 drugs (8 approved, 10 experimental)
- Walker-Warburg Syndrome
Drugs by status
Approved for this indication (6)
Phase 4 trials (4)
Phase 3 trials (6)
Phase 2 trials (12)
Phase 1 trials (8)
Organizations
Organization Involved with Phase 4 Indications (9)
Organization Involved with Phase 3 Indications (60)
- Biomarin
- Santhera
- Bristol-Myers Squibb
- Sarepta Therapeutics
- Eli Lilly
- Genzyme
- Pfizer
- Bioblast Pharma Ltd.
- FibroGen
- GlaxoSmithKline
- Italfarmaco
- PTC Therapeutics
- Otsuka
- National Center for Research Resources (NCRR)
- National Institute of Neurological Disorders and Stroke (NINDS)
- University of Pennsylvania
- University of Pittsburgh
- Cooperative International Neuromuscular Research Group
- Federal University of Minas Gerais
- University of Washington
- Katholieke Universiteit Leuven
- Newcastle University
- Ohio State University
- Universidad Complutense de Madrid
- University of Florida
- University of Kansas
- University of Milan
- University of Rochester
- University of Bern
- University of California, Davis
- +30 more
Organization Involved with Phase 2 Indications (61)
- Acceleron Pharma, Inc.
- Daiichi Sankyo
- Akashi Therapeutics
- Ipsen
- Wyeth
- Cumberland Pharmaceuticals
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Kobe University
- Mallinckrodt
- ReveraGen BioPharma, Inc.
- RSPR Pharma AB
- Sponsor GmbH
- Summit Therapeutics
- Capricor Inc.
- Nobel Pharmaceuticals
- Stem Cells Arabia
- Medical Research Council
- National Center of Neurology and Psychiatry, Japan
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- United States Army
- Acibadem University
- University of London
- Institut de Myologie, France
- Instituto Mexicano del Seguro Social
- Johns Hopkins University
- Kunming Medical University
- McGill University
- Northwestern University
- Thomas Jefferson University
- Laval University
- +31 more
Organization Involved with Phase 1 Indications (26)
- Asklepion
- Marathon Pharmaceuticals
- Medistem
- Roche
- Astellas
- BioLeaders Corporation
- EspeRare Foundation
- National Institutes of Health (NIH)
- Parent Project, Italy
- Harvard University
- Luigi Sacco University Hospital
- Medical University of Bialystok
- Neurogen Brain and Spine Institute
- University of Liverpool
- University of Manchester
- University of Messina
- University of Birmingham
- Celerion
- Gaziantep University
- Worldwide Clinical Trials
- Allergy and Asthma Consultants of Wichita, KS
- Aidan Foundation
- Parent Project Muscular Dystrophy
- The Myositis Association
- Epirium Bio Inc.
- Edgewise Therapeutics, Inc.
Organization Involved with Other Experimental Indications (8)