Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.

New effort to address rare genetic conditions that dramatically raise the risk of serious mental health challenges, including ...

Gene therapy has proved to be a safe long-term treatment for haemophilia B. The benefits of a single dose persist for at ...

A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...

With Nigeria home to the highest number of people living with sickle cell disease (SCD) globally, health experts have called ...

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases ...