An experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy ...

Please provide your email address to receive an email when new articles are posted on . Cardiomyopathy in Duchenne muscular dystrophy possesses a unique pathophysiology. Integrated wall stress can be ...

On Monday, the U.S Food and Drug Administration (FDA) lifted the clinical hold on Entrada Therapeutics, Inc. (NASDAQ:TRDA) ENTR-601-44. The agency issued the hold in December 2022. In November 2023, ...

Novartis had one eye on the struggles its peers have encountered in the muscular dystrophy space when it decided to acquire Kate Therapeutics, according to the Big Pharma’s CEO. The Swiss drugmaker ...

At the upcoming annual meeting of the American Society of Gene and Cell Therapy, scientists from Huidagene Therapeutics will share an update on the development of their ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. One key aspect of the journey for a person ...

Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...

A 28-year-old from Hull is planning to run the Manchester Marathon for a cause close to his heart. Nathan Benham is raising funds for Muscular Dystrophy UK. For over 60 years, the charity has built a ...