The boy was born with the rare disease that would gradually take away his cognitive and motor function without treatment.

Medical breakthroughs 2025 report highlights FDA approvals, gene therapies, and robotic telesurgery advancing patient care this year.

DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy CRO Market: Industry Trends and Global Forecasts, Till 2035 - Distribution by Type of Cell Therapy, Type of Genetic Modification, Stage of ...

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News: Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, ...

Bayer AG ($BAYRY) announced an update on their ongoing clinical study. Study Overview The PROGRESS-GT LOPD trial is an early-stage clinical study ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

Escandon is the founder and principal of DGBI Clinical Research and Ethics Consulting, a consultancy for the biotechnology industry. Caplan is Mitty professor of medical ethics at the NYU Grossman ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

Sarepta Therapeutics (NASDAQ:SRPT) fell 11% premarket on Monday after announcing the U.S. FDA has placed a clinical hold on its investigational gene therapy trials for limb girdle muscular dystrophy ...

MSK is the first hospital in New York City to offer gene therapy through its pediatric cancer program MSK Kids. Exagamglogene autotemcel (exa-cel) (Casgevy®) is a gene therapy for treating sickle cell ...