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 ...

Yet, despite the “stormy waters,” global sales of orphan drugs are projected to exceed $400 billion by 2032, according to a new report by Evaluate.

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 ...

If your child were critically ill, how far would you go to find answers?

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.

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

Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal ...

Months after promising help for “hundreds of thousands of kids,” the FDA approved the decades-old medicine for a disease documented in less than 50 people.

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 ...

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 ...

A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of treatments for rare diseases, adding political pressure on an agency that’s ...

Registration has opened for the online "Run for Kids with Rare Diseases" charity race, allowing participants to raise funds ...