This study was carried out on 9 patients with myotonic dystrophy and 8 with slowly progressive nonmyotonic muscular dystrophy admitted to the Neurology Service at Duke University Hospital and the ...
Conserving the reading frame of the protein has a determining factor in disease severity in many dystrophinopathies. Mutations in which the reading frame is conserved resulting in a functional amino ...
The muscular dystrophies represent disorders of progressive muscular degeneration and weakness. As a group, they exhibit clinical heterogeneity that reflects diverse molecular mechanisms responsible ...
The Food and Drug Administration Thursday approved the first gene therapy treatment for Duchenne Muscular Dystrophy, a progressive disorder that typically robs boys of their ability to walk around age ...
DR. JULIA BELL has contributed a useful monograph on muscular dystrophy in man to “The Treasury of Human Inheritance” series. Clinical and genetical data from more than 1,300 individuals exhibiting ...
SAN ANTONIO - A young college student finds strength in his family to cope with Duchenne muscular dystrophy, an inherited disorder of progressive muscular weakness that’s typically in boys. Corey ...
The U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...
Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial ...
Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...
The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...