An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

Treatment with gene therapy candidate BB-301 led to improvements in swallowing ability for the first three OPMD patients in a ...

The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

New Delhi, Over 200 parents from 26 states across India protested at Jantar Mantar on Wednesday with their children affected by Duchenne Muscular Dystrophy and Muscular Dystrophy , demanding urgent ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...