Track topics on Twitter Track topics that are important to you
Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene...
The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections...
Examples of Ophan Drug Indications include: Anaemia, sickle cell Cystic fibrosis (CF) Duchenne muscular dystrophy Glioma Graft vs host disease (GvHD) Hepatoma, liver cancer Hodgkin Lymphoma ...
Jude Almkhlef's family fled Syria in 2013. Now the 9-year-old, who has Ullrich congenital muscular dystrophy, is making great progress at Boston Children's.
Scientists have identified a unique form of congenital muscular dystrophy, characterised by short stature, intellectual disabilities and cataracts...
NewsProgressive muscle degeneration and weakness characterize Duchenne muscular dystrophy.
A pair of protein-inhibiting compounds is effective at slowing the progression of a form of muscular dystrophy in animal models, a group of University of Florida Health researchers has found. The comp...
aTyr Pharma Presents Analyses of Resolaris Phase 1b/2 Trial in Patients with Limb Girdle Muscular Dystrophy 2B and Facioscapulohumeral Muscular Dystrophy at the American Academy of Neurology 69th Annual Meeting
- Resolaris Demonstrated Favorable Safety Profile and Promising Signals of Clinical Activity - - Resolaris has FDA Fast Track and Orphan Drug Designation for Limb Girdle Muscular Dystrophy 2B (LG...
Monday, March 13th 2017 at 12:00pm UTC CAMBRIDGE, Mass.–(BUSINESS WIRE)– Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that it ...
A drugmaker fiven FDA approval to market a generic drug for muscular dystrophy has 'paused' rollout after an outcry over its $89,000-a-year price tag.
Researchers at the National Institutes of Health's National Center for Advancing Translational Sciences (NCATS) and the University of Nevada, Reno School of Medicine (UNR Med) have demonstrated that a...
We report a patient with a typical phenotype and clinical history of Duchenne muscular dystrophy who is currently 53 years old. Because of improvements in cardiopulmonary care, there has been a great ...
In Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), interventions reducing the progression of myocardial disease could affect survival.
In adult patients with Duchenne muscular dystrophy (DMD) life-threatening constipation has been reported. Since gastrointestinal function in DMD has not been rigorously studied we investigated objecti...
This article describes the clinical characteristics, diagnosis, molecular pathogenesis, and treatment of facioscapulohumeral muscular dystrophy (FSHD).
Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA) causes significant disability and progressive functional impairment. Readily available instruments that assess functionality, especi...