Solid Biosciences (SLDB) reported updated “positive” interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. The new data, including additional muscle biopsy, serum ...

In exploring barriers in rare disease research and drug development, experts highlight a lack of robust models, limited trial populations, fragmented global expectations, and production bottlenecks as ...

Recent transactions make clear that large pharmaceutical companies now view rare disease as a core area for sustained growth, rather than episodic deal-making. BioMarin’s acquisition of Amicus ...

Barclays 28th Annual Global Healthcare Conference March 12, 2026 8:30 AM EDTCompany ParticipantsAmy Parison - Senior VP ...

REGENXBIO (NASDAQ:RGNX) outlined a slate of near-term clinical catalysts and provided financial updates during its ...

Explore how proteomics in biomarker discovery accelerates diagnostic assay development and improves clinical validation.

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...

"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.

The first cell rejuvenation therapy is set to enter clinics, testing if aging cells can be reset to a younger, healthier ...

A novel bioengineering strategy utilizing peptide display technology on the AAV1 capsid has successfully generated next-generation viral vectors with ...

With clinical data beginning to emerge, big pharma is signaling its belief that in vivo CAR generation is the next immunotherapy breakthrough.

Rocket Pharmaceuticals, Inc. ( RCKT) TD Cowen 46th Annual Health Care Conference March 2, 2026 11:10 AM EST ...