Zevra Therapeutics, Inc. (NASDAQ:ZVRA), a biopharmaceutical company focused on developing and commercializing novel medicines for rare diseases, has recently achieved a significant milestone with ...

Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

Zevra Therapeutics, Inc. (NASDAQ:ZVRA), a biopharmaceutical company focused on developing and commercializing novel medicines for rare diseases, has recently achieved a significant milestone with the ...

CAMP4 Therapeutics raises $75 million in IPO, advances RNA therapies for rare diseases, and partners with BioMarin. Analysts ...

Rare Pediatric Disease Designation is granted by the FDA for serious ... that could be utilized by the Company or potentially sold to another company for its use. About Actuate Therapeutics, Inc.

However, analysts note potential flexibility from the FDA regarding rare diseases, which could benefit PTC Therapeutics' approval strategies. PTC Therapeutics is well-positioned in the rare disease ...

However, analysts note potential flexibility from the FDA regarding rare diseases, which could benefit PTC Therapeutics' approval strategies. PTC Therapeutics is well-positioned in the rare ...

We recently published a list of 10 Best Multibagger Stocks To Buy Heading into 2025. In this article, we are going to take a ...

Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare ...

DUBLIN--(BUSINESS WIRE)--Oranda Therapeutics, a new Irish headquartered rare disease company with an innovative, fast to market, commercial approach, today announces the launch of their Series A ...