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 ...
EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and ...
Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...
Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...
Spain’s ARTHEx Biotech has developed a dual-mechanism approach to treating the most common adult-onset muscular dystrophy, myotonic dystrophy type 1, setting it apart in a burgeoning blockbuster ...
Investigational delpacibart etedesiran (del-desiran) showed promise in the phase I/II MARINA trial of myotonic dystrophy type 1 (DM1), a rare progressive disorder caused by a toxic gain-of-function ...
Muscular dystrophy is a group of inherited conditions that lead to progressive weakening of voluntary muscles and, in some cases, impact the heart and other organs. There are nine major types of ...
WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that screening is underway in Study ...
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