Children's National Hospital experts developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit ...

Medical experts have developed a new pre-clinical gene therapy for a rare disorder, known as limb-girdle muscular dystrophy (LGMD) 2B, that addresses the primary cellular deficit associated with this ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today ...

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 ...

Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing ...

– U.S. FDA has confirmed that screening and dosing may proceed in Study SRP-9005-101 for LGMD2C/R5 – Enrollment and dosing completed in Study SRP-9004-102 for LGMD2D/R3 – Data expected for SRP-9003 ...

-- Sarepta strengthens position as a leader in gene therapy; expands rare disease franchise -- -- The program is designed, if successful, to offer first-ever corrective treatments for five distinct ...

– Sarepta’s unrivaled portfolio of investigational gene therapies for LGMD offers the potential to address six LGMD subtypes, which together represent more than 70% of all known LGMDs CAMBRIDGE, Mass.

Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing ...

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 ...