To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT), a commercial-stage biotechnology company with a market capitalization of $446.4 million specializing in mRNA therapeutics and vaccines, stands at a ...

SEED Therapeutics announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease and Orphan Drug designations to ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...

The early stage company is developing a new approach to gene editing to treat rare genetic diseases. It does this using mRNA, ...

Learn more about whether Avidity Biosciences, Inc. or TG Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

This case study explores a rare overlap of hidradenitis suppurativa and porokeratosis, as well as highlights the unique ...

Cofounded by Dr. Siddhartha Mukherjee and Reid Hoffman, Manas AI leverages proprietary AI, generative computational chemistry ...