Union Minister H D Kumaraswamy has assured assistance for a two-year-old child suffering from a rare genetic disorder after ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Brooklyn Nets radio play-by-play voice Chris Carrino Sr. was honored by the Naismith Basketball Hall of Fame with a Curt Gowdy Media Award.

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 ...

FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who have been diagnosed with FA and ...

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 ...

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

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