Muscular Dystrophy, Duchenne
An X-linked recessive muscle disease caused by an inability to synthesize DYSTROPHIN, which is involved with maintaining the integrity of the sarcolemma. Muscle fibers undergo a process that features degeneration and regeneration. Clinical manifestations include proximal weakness in the first few years of life, pseudohypertrophy, cardiomyopathy (see MYOCARDIAL DISEASES), and an increased incidence of impaired mentation. Becker muscular dystrophy is a closely related condition featuring a later onset of disease (usually adolescence) and a slowly progressive course. (Adams et al., Principles of Neurology, 6th ed, p1415)
Drugs by status
Approved for this indication (9)
Phase 4 trials (4)
Phase 3 trials (26)
- sodium nitrate
- prednisone
- drisapersen
- edasalonexent
- l-glutamine
- idebenone
- citrulline
- nebivolol
- pamrevlumab
- bms-986089
- ataluren
- sitagliptin
- pizuglanstat
- spironolactone
- allogeneic cardiosphere-derived cells
- creatine
- l-epigallocatechin
- eplerenone
- metoprolol
- tamoxifen
- bisoprolol
- lisinopril
- ubiquinone
- fordadistrogene movaparvovec
- wve-210201
- sgt-003
Phase 2 trials (48)
- methylprednisolone
- ace-031
- corticotropin
- theophylline
- Poloxalene
- pro044
- canakinumab
- pemirolast
- pro053
- ds-5141b
- ezutromid
- icosapent ethyl
- ifetroban
- mecasermin
- Arbekacin
- myo-029
- pf-06252616
- pro045
- bmn053
- stem cells
- pentoxifylline
- umbilical cord mesenchymal stem cells
- autologous skeletal myoblast
- raav1.cmv.hufollistatin344
- raavrh74.mck.galgt2
- autologous mesenchymal stem cells
- sildenafil
- halofuginone
- fish oil
- sgt-001
- +18 more
Phase 1 trials (18)
Organizations
Organization Involved with Phase 4 Indications (7)
Organization Involved with Phase 3 Indications (52)
- Biomarin
- Santhera
- Bristol-Myers Squibb
- Eli Lilly
- Genzyme
- Pfizer
- 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
- University of California, Los Angeles
- University of Colorado, Denver
- +22 more
Organization Involved with Phase 2 Indications (55)
- 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
- 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
- Northwestern University
- Thomas Jefferson University
- Laval University
- University of Cincinnati
- University of Copenhagen
- +25 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
- 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
- Hebrew University
- Aidan Foundation
- Parent Project Muscular Dystrophy
- The Myositis Association
- Epirium Bio Inc.
- Edgewise Therapeutics, Inc.
Organization Involved with Other Experimental Indications (4)