Care hurdles now add to the disease burden faced by U.S. sickle cell patients, despite the promise of emerging options like gene therapy.

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.

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 ...

A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

A team of University of Kentucky researchers has uncovered a surprising clue in the battle against Alzheimer's disease that could help doctors predict, and ultimately prevent, a common side effect of ...

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

Proof-of-concept trial in a single patient shows that cells can survive transplantation without immunosuppression ...

IT’S 1123. MORE THAN 300 MILLION PEOPLE WORLDWIDE ARE LIVING WITH A RARE DISEASE, AND MOST STILL DON’T HAVE EFFECTIVE TREATMENTS. BUT BREAKTHROUGHS IN CELL AND GENE THERAPY ARE SLOWLY CHANGING THAT.

Dustin Vidrine's sight is disappearing — shrinking, really. He has retinitis pigmentosa, a rare, inherited eye disease that runs in his family. By the time he was in his 20s, he noticed that his ...

The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung ...

Feb 9 (Reuters) - Regenxbio (RGNX.O), opens new tab said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for a rare disease called Hunter syndrome, citing ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by the FDA. In rejecting RGX-121, the FDA raised concerns about its clinical ...