We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...
Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...
The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years and ...
A preclinical study led by the Germans Trias i Pujol Research Institute (IGTP), in collaboration with the Institut de ...
With the writing apparently on the wall, PTC Therapeutics has called off its latest bid for FDA approval of its Duchenne muscular dystrophy (DMD) drug Translarna. | Late Thursday, PTC revealed that ...
Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...
A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...
Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that can lead to the slow deterioration of muscles, predominantly in the face, shoulder blades, and upper arms. Facioscapulohumeral ...
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