Ultragenyx said that the trial of the gene therapy will continue while data from the second primary endpoint is collected.

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

Ultragenyx Pharma (RARE) stock is in focus as gene therapy DTX301 met its main goal in a late-stage trial for the most common ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...

Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeksClinically important changes ...

What if the darkness that enveloped a child's world suddenly… vanished? What if hope, once a whisper, roared into life with a ...

Oppenheimer initiated coverage of Ocugen (NASDAQ:OCGN) with an Outperform rating and a $10 price target on Wednesday, ...

Biogen’s salanersen is now heading into three Phase III trials in SMA.

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...