While Duchenne Muscular Dystrophy is typically inherited, about one-third of cases arise from new mutations that occur spontaneously (with no family history). Males, who inherit one X chromosome from ...

Boston, Massachusetts--(Newsfile Corp. - April 29, 2025) - Tevard Biosciences, Inc., a privately held biotechnology company pioneering tRNA-based therapies to cure a broad range of genetic ...

We still have maybe 15% or so of people who come to our clinic that we're not able to establish the cause of their muscular dystrophy ... and new discovery in the genetic space for those individuals.

Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

From STAT’s Adam Feuerstein: The FDA informed Capricor Therapeutics that a meeting of outside experts will be convened to ...

SRPT), the leader in precision genetic medicine for rare diseases, today shared updates from its clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3 ...

Scientists from The Hospital for Sick Children (SickKids) and University of Las Vegas Nevada (UNLV) have uncovered a genetic link ... (NIH), Myotonic Dystrophy Foundation, Muscular Dystrophy ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy ... with the X-linked muscle-wasting ...

and a rare genetic condition called myotonic dystrophy type 1 (DM1). The study, published today in Nature Neuroscience, suggests that while ASD has previously been characterized by a loss of gene ...