New analyses out to 24-months showed improvement in heart and lung function compared to expected declines in DMD natural history -- Data expand ...

HARMONIA trial will assess multi-system efficacy, safety and tolerability of z-basivarsen in DM1 -- 48-week trial will enroll approximately 150 ...

Early diagnosis of rare diseases in children can significantly improve treatment outcomes and quality of life. Learn symptoms, challenges, and why early detection is crucial for managing pediatric ...

As policies promise support and courts intervene, thousands of rare disease patients remain trapped between soaring drug ...

Vinay Prasad’s exit — his second from the agency in less than a year — comes after sharp criticism of his handling of drug ...

Doctors warn that Bulgaria’s healthcare system still lacks a sustainable framework for genomic diagnostics, patients are paying the price ...

Experts call for early diagnosis, prophylaxis in hemophilia, improved MS policy frameworks, genomic testing access and ...

Pratteln, Switzerland, February 27, 2026 - Santhera Pharmaceuticals (SIX: SANN) today announces that the Spanish Interministerial Commission on Prices of Medicines, has proposed to include AGAMREE (va ...

Phil Farley has survived 25 years with an untreatable disease that typically kills it victims within three years of diagnosis ...

Thanks to advances in imaging and diagnostic technologies, clinicians can now detect many genetic disorders in the womb, ...

Dr. Vinay Prasad drew criticism for overriding career scientists and rejecting drugs by companies seeking agency approval.

Rokhaya Ndiaye Diallo, is raising the alarm over the financial burden of diagnosing and treating rare diseases in Senegal and is calling for a dedicated national plan.The diagnosis and management of r ...