CAMBRIDGE, Mass., April 08, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today stated that its previously announced ...

BridgeBio Pharma, Inc. announced it will host an investor webinar on July 11, 2025, at 8:00 am ET, featuring Dr. Matthew Wicklund from the University of Texas Health Science Center. The webinar will ...

PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced ...

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 ...

DUBLIN--(BUSINESS WIRE)--The "Limb Girdle Muscular Dystrophy (LGMD) - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering. Limb Girdle Muscular Dystrophy (LGMD) ...

Sarepta Therapeutics (NASDAQ:SRPT) fell 11% premarket on Monday after announcing the U.S. FDA has placed a clinical hold on its investigational gene therapy trials for limb girdle muscular dystrophy ...

It's not easy to distinguish between the dozens of subtypes of limb-girdle muscular dystrophy - a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes ...

The advent of gene therapies, which can target specific variants, means pinpointing the genetic roots of each patient’s disease has taken on new importance, researchers said. A team of researchers ...

Please provide your email address to receive an email when new articles are posted on . Survey data provided insight into the symptomatic burden of limb girdle muscular dystrophy, according to study ...

The most common limb-girdle muscular dystrophy subtype in Italy, Spain, Turkey, Russia, Brazil and Australia is calpainopathy (limb-girdle muscular dystrophy type 2A), while dystroglycanopathy ...

Daughter Verena shares her personal story of living with Limb-girdle type 2A muscular dystrophy. The progressive disease affects muscles around the shoulders, limbs, hips, and thighs, making simple ...