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 ...

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 ...

Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...

Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced an update on the building momentum in the Company’s launch of FDA-approved ZEVASKYN (prademagene zamikeracel), a first-of-its-kind, autologous ...

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 ...

About five months after U.S. Department of Health and Human Services Secretary Robert F. Kennedy Jr. | About five months after U.S. Health Secretary Robert F. Kennedy Jr. touted leucovorin as “an ...

More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

With clinical data beginning to emerge, big pharma is signaling its belief that in vivo CAR generation is the next immunotherapy breakthrough.

Researchers have uncovered a potent genetic mechanism that could revolutionize cancer therapy. The immune system naturally generates a molecule known as IL-15, which serves as a defender that triggers ...

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 ...

Now, Chinese scientists have developed a kind of "double-sided tape" that helps the body's own soldiers — engineered immune cells — hold onto cancer cells more tightly, making treatment far more ...

There is a hidden genetic switch in the DNA of every cancer patient, a sequence capable of determining whether the most advanced therapy of the moment immunotherapy will be a blessing or a ...