GRIN Therapeutics & Angelini Pharma ink exclusive collaboration to develop and commercialize radiprodil outside North America: New York Wednesday, May 28, 2025, 15:00 Hrs [IST] GR ...

US FDA places clinical hold on Rocket Pharma's gene therapy phase 2 trial of RP-A501 to treat Danon disease: Cranbury, New Jersey Wednesday, May 28, 2025, 14:00 Hrs [IST] Rocket P ...

A girl was diagnosed with NARS1 after having difficulty speaking may finally have a chance to live. For nearly a decade, ...

The first systematic genome-wide association study for pseudogout has identified two genes strongly associated with disease progression.

What they didn’t know was that a dangerous mutation was quietly being passed from one child to the next. It started with two ...

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

The sperm of a man carrying a rare genetic mutation linked to cancer was used to conceive scores of children across Europe, ...

Scientists have made great progress in understanding what makes some people live past 100 while others don’t make it to 80. It turns out the secret isn’t just good genes. In fact, genes may play less ...

Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. The biotechnology said Tuesday that the Food and Drug Administration granted the designation ...

A new study has shown that lowering levels of a protein called EMC10 can restore brain function and memory in models of 22q11.2 Deletion Syndrome, pointing to a potential treatment approach.

The test applies to potentially thousands of different diseases, and it can even detect new ones by providing the evidence ...