Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...

More than 150,000 people are affected by muscular dystrophy in the UK - 30,000 suffer directly as a result of their condition but a further 120,000 people including families and carers have had ...

Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, ...

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned ...

Title: RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy: Interim clinical data (O75 ... Enhanced therapeutic potential of a microdystrophin with an extended ...

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' Zolgensma® for children with spinal muscular atrophy. Designed to be one-time treatments ...

If you’re living with Duchenne muscular dystrophy (DMD), planning ahead can help support the success of future education, employment, and housing efforts. Duchenne muscular dystrophy (DMD ...