The University at Albany and Virginia Commonwealth University were awarded a $6.5 million NIH grant to establish a federal ...

Givinostat is an oral histone deacetylase (HDAC) inhibitor approved by the FDA in 2024 to treat Duchenne patients ages 6 years and older. Pathologic upregulation of HDAC occurs in DMD, leading to ...

BridgeBio said an experimental therapy for a rare form of muscular dystrophy succeeded in a key trial, potentially setting up ...

His parents Stephen and Jenny have asked Sheffield Children's NHS Foundation Trust to approve the use of drug givinostat, ...

One of those things is the CureDuchenne clinic in Denton. It provides care to underserved and uninsured people with Duchenne. "I think it's good that we're trying to find the cure," Shep said.

Data from the Phase Ia/b study demonstrated that SAT-3247 was well-tolerated and safe with a desirable PK profile.

Already sky high after the success of its limb-girdle muscular dystrophy therapy, reported on Monday, BridgeBio’s shares rose nearly 8% more as encaleret balanced calcium levels in patients with a ...

New interim data from a Phase III trial puts the company on track to file for FDA approval next year in an indication that ...

Increasing your stamina benefits you long after you complete your last rep or final mile, too. In fact, maintaining endurance ...

Charlie Zerzan refuses to let SMA define him, using wheelchair basketball to chase championships and inspire others.