we propose that there are at least 4 clinically and genetically distinct forms of X-linked muscular dystrophy. IV-6 (A.C.), born in 1927, was not examined. He died of unrelated congenital heart ...
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the ...
Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire ...
Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...
The Muscular Dystrophy Association (MDA)’s Kickstart program has announced receipt of both U.S. orphan drug and rare pediatric disease designations in support of work for congenital myasthenic ...
Thin Ribs on Chest X-Ray—a Sign Pointing the Way to the Diagnosis of Congenital Myotonic Dystrophy
In addition, the infant exhibited pronounced muscular hypotonia (“floppy ... This strengthened the suspicion of congenital myotonic dystrophy type 1 (DM1) in the newborn. Autosomal dominant ...
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