Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum ...

Leerink analyst Mani Foroohar raised the firm’s price target on Regenxbio (RGNX) to $20 from $16 and keeps an Outperform rating on the shares.

Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

A discovery in mice reveals why fasting enhances a type of breast cancer treatment — a hormone-signalling pathway and gene-expression changes have key roles. Stephen D. Hursting is in the Department ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.

Last 28 March 2025, the European Commission decided not to renew the conditional marketing authorisation for Translarna (ataluren) in Duchenne’s disease due to lack of efficacy after 11 years in the ...

Jada Bafus told Page Six that she and the actress quickly came to realize it was a case of “two triggered mothers” having a ...

After learning devastating news about our young son, we needed to feel connected to friends and family, not just exist beyond ...

A family raising money to fund a suitable home for their son is among those driving a revival in honesty boxes.