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

Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.

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

Topline data were announced from a phase 3 trial evaluating DTX301 (avalotcagene ontaparvovec) for the treatment of OTC deficiency.

Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.

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

March 12 (Reuters) - Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a late-stage trial to better control ammonia levels linked to a rare inherited disorder ...

After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question ...

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

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 ...

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...