Salanersen, a treatment candidate for spinal muscular atrophy (SMA) now in clinical testing, is safe and was shown to stabilize or improve motor function over at least one year of follow-up in ...

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 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 ...

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 ...

Hemgenix gene therapy for hemophilia B helped many patients, even those with pre-existing anti-AAV5 antibodies. It increased factor IX levels, significantly reduced bleeding, and lowered the need for ...

Knoepfler is a professor of cell biology and human anatomy at UC Davis School of Medicine. At a Food and Drug Administration cell and gene therapy (CGT) roundtable last June, most speakers pushed for ...

Forbes contributors publish independent expert analyses and insights. A patient in a late-stage gene editing trial for a rare heart condition died from fatal liver complications after receiving an ...