– 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 ...

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 ...

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 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 ...

Sarepta Therapeutics has come under pressure following the deaths of three patients using its treatments.

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 ...

Friends and family of Sophie Hutchison, who was diagnosed with limb girdle muscular dystrophy at 13, will take part in this ...

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...

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 ...

To continue reading this content, please enable JavaScript in your browser settings and refresh this page. Preview this article 1 min The search for a treatment for ...