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

Two key pilot programmes - Support for clinical Trials Advancing Rare disease Therapeutics (START), launched in 2023, and Collaboration on Gene Therapies (CoGenT), introduced in early 2024 - were ...

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

"This type of gene editing should also allow for the treatment of diseases whose origin is not genetic or whose origin cannot ...

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 .

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

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

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

India should eliminate tax on drugs for cancer treatment and for chronic and rare diseases, the chairperson of major ...

“Nippon Shinyaku has an impressive and established track record of commercializing rare disease therapies in ... pro-inflammatory cytokine IL-18, the therapeutic target of Tadekinig alfa.

AB2 Bio signs U.S. option and licensing agreement with Nippon Shinyaku for Tadekinig alfa for an ultra-rare autoimmune disease AB2 Bio Ltd Sun, Jan 26, 2025, 11:30 PM 5 min read ...