STAT

FDA unveils rules for bespoke gene therapies, predicting flood of rare disease applications

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...
Wall Street Journal

FDA: How We’re Approving More Drugs to Treat Rare Diseases

Rare diseases aren’t so rare when you add them up. They affect roughly 1 in 11 people, which comes to about 30 million Americans. Rare diseases are more common than asthma and Type 1 diabetes combined ...
The Hill

FDA proposes new treatment approval pathway for ultrarare diseases

The Food and Drug Administration (FDA) announced a new proposal Monday for flexible drug approval pathway treatments addressing ultrarare diseases. The FDA unveiled draft guidance on a proposed ...
CNN

FDA proposes new system for approving customized drugs and therapies for rare diseases

Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the ...
CNBC

FDA proposes new system for approving customized drugs and therapies for rare diseases

Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the ...
Science Daily

AI breakthrough could replace rare earth magnets in electric vehicles

Scientists at the University of New Hampshire have unleashed artificial intelligence to dramatically speed up the hunt for next-generation magnetic materials. By building a massive, searchable ...
Becker's Hospital Review

FDA unveils pathway for ultra-rare disease therapies

The FDA has released draft guidance outlining an approval pathway for individualized therapies aimed at ultra-rare diseases, building on a framework first outlined by agency leaders in November. The ...
Reuters

US FDA proposes framework to speed rare disease gene therapy approvals

WASHINGTON, Feb 23 (Reuters) - The U.S. Food and Drug Administration proposed on Monday a new framework to speed approvals of personalized treatments for rare and life-threatening genetic diseases, ...
The American Journal of Managed Care

FDA Proposes New Approval Pathway for Individualized Ultra-Rare Disease Therapies

FDA proposes using a well-supported mechanistic rationale plus natural history comparators to generate substantial evidence when randomized trials are infeasible for ultra-rare populations.
NPR

The FDA creates a quicker path for gene therapies

The Food and Drug Administration Monday unveiled the details of a new policy designed to make it easier and quicker for patients with very rare diseases to get cutting-edge treatments. The new ...
Hosted on MSN

Is USA rare earth stock a once-in-a-decade rare earth opportunity?

USA Rare Earth has yet to begin either commercial production of rare earth magnets or elements from the Round Top deposit. Its focus on high-value heavy rare earth elements gives it significant upside ...
Bloomberg L.P.

Brazil, India seal rare earth deal amid global supply strains

Connecting decision makers to a dynamic network of information, people and ideas, Bloomberg quickly and accurately delivers business and financial information, news and insight around the world ...