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

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

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

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.

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

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.

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 ...

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 ...

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