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

As Malaysia commemorates National Rare Disease Day and marks the launch of the National Policy for Rare Diseases, long-term, ...

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 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 ...

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 ...

Rare diseases are complicated and communicating reliable, accessible health information around them can be even more complicated. Some conditions like RPI deficiency or Aicardi syndrome affect less ...

A new artificial intelligence system called DeepRare has beaten experienced rare-disease physicians at their own specialty, ...

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.

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

Global rare disease policy shifts are reshaping orphan drug development, with major implications for equitable access in the UK, EU and US.

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 ...

Nick Jonas mourns childhood friend Maya Kibbel's death at age 30 Maya Kibbel battled Wilson's disease, a rare genetic disorder Jonas family and fans remember Maya as a close family member Did our AI ...