In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

Amino acid balance could play a role in SMA, as a study showed acids able to influence nervous system function were ...

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 ...

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

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

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 ...

“We were referred to a paediatrician who put him on reflux medication. However, things were getting worse,” Ellie said. “He ...

Chugai Pharma’s Evrysdi tablets 5mg receives Japanese approval to treat spinal muscular atrophy: Saturday, March 29, 2025, 11:00 Hrs [IST] Chugai Pharmaceutical Co., Ltd. announ ...

Neuromuscular diseases encompass a wide range of disorders characterized by progressive muscle weakness and dysfunction, resulting from abnormalities in the ...