Cholesterol meds—taking by millions of Americans every day—often come with a lifelong prescription, adding out-of-pocket ...

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

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...

Current gene therapies to treat sickle cell disease are complex, time-consuming, and are sometimes linked to serious side effects like infertility or blood cancer. To address these challenges, Johns ...

Delivering the hormone leptin directly to the brain through gene therapy aids weight loss without the significant side effect of bone loss, according to new collaborative research from Oregon State ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

Itvisma contains the active ingredient in Zolgensma approved in 2019 for children younger than 2 years but is reformulated ...

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

FDA cleared Itvisma as the first gene-replacement therapy available to people 2 and older with the rare muscle-weakening ...