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

A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

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.

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

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 ...

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 ...

The FDA is already under fire from lawmakers for recent delays and rejections of new drugs for rare diseases.

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 Caribbean Community (CARICOM) grouping is calling for stronger surveillance system amid on going efforts to deal with the impact of rare ...

New Czech rare disease strategy prioritises faster diagnostics, multidisciplinary collaboration and the introduction of care coordinators ...