PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls

New Insights into Muscular Dystrophy Treatment Unveiled by Scientists

A groundbreaking study by researchers at the USC Dornsife College of Letters, Arts and Sciences has illuminated the complex mechanisms behind the formation of tiny protein clusters in human cells. This research holds significant potential for advancing treatments for Emery-Dreifuss muscular dystrophy (EDMD), a rare genetic disorder marked primarily by severe muscle weakness and various heart complications. With the interplay of advanced imaging …

“My life changed forever when I was diagnosed..." Running the London Marathon with muscular dystrophy - London Post

In 2018, Lew Mathias, 29, an engineering manager from Clapham, London was diagnosed with Myotonic Dystrophy Type 1, a rare muscle wasting condition. Determined to prove to himself, as well as to his friends and family, that he can overcome challenges and achieve extraordinary things despite his condition, he has signed up to take part in the London Marathon 2025. Lew is running in support of Muscular Dystrophy UK, the leading charity for the 110…

PepGen pauses Phase II DMD trial to review safety data

PepGen has previously faced an FDA clinical hold on the investigational new drug application of PGN-ENO51 in the Phase II CONNECT2 trial.The post PepGen pauses Phase II DMD trial to review safety data appeared first on Clinical Trials Arena.

PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls

On Tuesday, PepGen Inc. (NASDAQ:PEPG) announced its voluntary decision to temporarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients …

JCI Impaired hydrogen sulfide biosynthesis underlies eccentric contraction–induced force loss in dystrophin-deficient skeletal muscle

Eccentric contraction–induced (ECC-induced) force loss is a hallmark of murine dystrophin-deficient (mdx) skeletal muscle that is used to assess efficacy of potential therapies for Duchenne muscular dystrophy. While virtually all key proteins involved in muscle contraction have been implicated in ECC force loss, a unifying mechanism that orchestrates force loss across such diverse molecular targets has not been identified. We showed that correct…

Edgewise’s recent business highlights from 2024 include advances in their clinical trials in both Duchenne and Becker

CureDuchenne provided early funding to Edgewise Therapeutics, and we are pleased with their progress as recently shared in their fourth quarter and full year 2024 reports.  In Becker muscular dystrophy, they have completed enrollment in their pivotal GRAND CANYON trial, and are advancing a Phase 2 open label extension trial (MESA) for long-term follow up.  In Duchenne, both the LYNX and FOX Phase 2 trials are ongoing.  The programs in both Duche…