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, ...

Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

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.

FLC is a rare liver cancer with unique genetic traits, often misdiagnosed until advanced. New research and a clinical trial ...

Icelandic genomics company Arctic Therapeutics has closed a €26.5 million (US$27.6 million) series A, enabling it to assess if its lead drug AT-001, designed to treat a rare inherited amyloid disease, ...