A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

Wisconsin Sen. Ron Johnson is asking to see several of the complete response letters that have been delivered of late and also told Bloomberg that he may call on top FDA leaders to testify before the ...

Spruce Biosciences has named Dale Hooks chief commercial officer, securing a leader with experience launching 21 products as the company heads toward a filing for its first FDA approval.

The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease ...

The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same ...

Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only ...

The U.S. Senate unanimously approved a bipartisan resolution declaring Feb. 28, 2026, as Rare Disease Day, aiming to expand ...

Torie Bosch is the First Opinion editor at STAT. Celena Lozano’s son Benny, who turned 5 in November, loves trains, trucks — anything that goes. He also has a rare disease, PURA syndrome. Earlier this ...

The survival rate in infants with severe combined immunodeficiency, a rare hereditary disease that prevents the body from ...

This article is part of a Rare Disease series sponsored by DeepIntent. With Rare Disease Month wrapping up and Rare Disease Day taking place on Saturday, MM+M is celebrating a handful of campaigns ...

CG Life, a medical marketing agency focused on rare disease and precision medicine, today launched 24/364, a cultural brand ...