A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

Treating our first patient is a proud moment for Abeona and a testament to the tireless resolve of our team,” said Vish ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

The FDA's approval of SMA treatment Itvisma marks an "important moment" for the SMA community, a Novartis executive says.

Born in Morristown, N.J., with virtually no immune system, Cora was diagnosed with severe combined immunodeficiency, a rare ...

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in ...

A new gene test might predict skin side effects from a common gout medication Two genes combined can account for side effect risk in more than 80% of U.S. gout patients The current test uses only one ...

Moment of Clarity has released a new educational resource titled “What Are the TMS Therapy Side Effects?” that provides an evidence-based look at the safety, tolerability, and efficacy of transcranial ...

An experimental MeiraGTx gene therapy that has clinical data showing reversal of blindness in children born with a rare, inherited eye disorder is heading to Eli Lilly. According to the terms of the ...

The FDA has put a clinical hold on a Tenaya Therapeutics gene therapy study while the biotech works to standardize activities related to the immunosuppression regimen across trial sites. Tenaya said ...