The New England Journal of Medicine23d
X-Linked Pseudohypertrophic Muscular Dystrophy with a Late Onset and Slow Progression
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 ...
BioWorld8d
In muscular dystrophies, gene therapies race the clock
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding ...
40 years of Bhopal gas tragedy: How the disaster affected people even before their birth
Forty years ago, on the intervening night of December 2-3, a terrible tragedy struck Bhopal as highly toxic methyl isocyanate ...
FierceBiotech13d
Novartis, assessing peers’ muscular dystrophy failures, pens $1.1B deal for gene therapy biotech
Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire ...
BioWorld1mon
MDA’s Kickstart program announces orphan drug designation for congenital myasthenic syndrome
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 ...