Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne, commemorated its 30th ...

Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Trump fingers Robert F. Kennedy Jr. to lead the HHS, lupus and ATTR-CM dominate headlines this week, bluebird bio has a cash gap to leap and RegenxBio eyes Sarepta in Duchenne muscular dystrophy.

Capricor Therapeutics (CAPT) stock in focus as company receives Orphan Drug and ATMP designations from the EU regulator for its lead drug deramiocel. Read more here.

Throughout 2024, the FDA issued new drug approvals and expanded treatment indications across various rheumatologic diseases.

Disabled people give testimony in phase three of the Covid inquiry. The latest part of the Covid inquiry, which looks at the impact of the pandemic on the NHS, has heard powerful evidence from ...

For example, there is an 11-year-old boy with Duchenne muscular dystrophy, which causes progressive muscle degeneration. He ...

Bank of America Securities analyst Alec Stranahan maintained a Buy rating on RegenXBio (RGNX – Research Report) yesterday and set a price ...

Oxford-based SynaptixBio, which is developing an antisense oligonucleotide (ASO) to ‘silence’ the expression of a single mutated gene, says the ASO market is set to grow dramatically, largely because ...

Mutual of America Capital Management LLC cut its position in shares of Sarepta Therapeutics, Inc. (NASDAQ:SRPT – Free Report) ...

Columnist Shalom Lim tells an international conference about his experiences with inclusion during his education in Singapore ...