Leader of Opposition Suvendu Adhikari donated Rs 10 lakh from his salary towards the treatment of a girl from West Bengal's ...

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

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...

Japanese drugmaker Chugai (TYO: 4519) has obtained regulatory approval from the Ministry of Health, Labor and Welfare (MHLW) ...

A new tablet formulation of SMA treatment Evrysdi (risdiplam) may give patients more "freedom and independence," a Genentech ...

In less than two weeks, the country came together and collected over KGS 510 million (USD 5.9 million), displaying unprecedented unity and generosity.

Gene therapy treatment gives Baby Kiama a lifeline, saving him from world’s most expensive drug that he needed to slow down a ...

Here is our list of the 11 Best Gene Therapy Stocks to Buy According to Analysts.

A majority of people have supported the urgent implementation of early screening for a genetic disease that causes severe ...

“During the wait for the results, things deteriorated. He was choking constantly, not going to the toilet for days and his ...

The family vehicle helps their teen daughter, who is in a wheelchair, drive more easily. Video from the car caught the act ...