Hopkins neuroscientist Richard Huganir is close to finding a potential life-changing treatment for kids with SYNGAP1-related disorders, but cuts to federal funding could delay or halt the progress ...

It's a slow-moving train wreck," Mike Graglia says about his 12-year-old son Tony's rare genetic disease with no cure.Caused by a tiny fluke of ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...

following her seven-year-old son Archie’s muscular dystrophy diagnosis. The rare condition currently affects 110 boys in Ireland, with no cure. However, specialist gene therapy is available in ...

Duchenne muscular dystrophy (DMD), recognised by the World ... However, emerging genetic therapies such as exon skipping and gene therapy offer hope --though at a cost that remains out of reach ...

I had the pleasure of attending a Netflix event around the WoW documentary The Remarkable Life of Ibelin last year—one that was attended by folks from Muscular Dystrophy UK, a charity that helps ...

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD ... a "best-in-class therapy designed to suppress ...

Just a few weeks after Avidity Biosciences made a stir with new data on its experimental therapy for Duchenne muscular dystrophy, the biotech has closed a public offering that raised an impressive ...

Boccia's appeal and rising popularity comes from its showcase as a Paralympic sport that can be enjoyed by people of all ages ...

Tristan Boedts, 28, has limb girdle muscular dystrophy (LGMD), which causes muscles to weaken over time, but has taken on intrepid adventures, including hiking to Mount Everest's Base Camp.

Apr. 28, 2025 — In the past 20 years, the average rate of obesity among adults in the United States has risen by approximately 30 percent, but the rate of those with ... Why Our Waistlines ...