The kinase inhibitor is now approved for adults and children aged ≥ 2 years with neurofibromatosis type 1 who have ...

The FDA has approved Evrysdi in tablet form for patients with spinal muscular atrophy aged 2 years and older who weigh more ...

CARDIFF, UK / ACCESS Newswire / February 12, 2025 /Biodexa Pharmaceuticals PLC. (NASDAQ:BDRX), a clinical-stage ...

Adult and pediatric patients with neurofibromatosis type 1 who have symptomatic plexiform neurofibromas that are not amenable ...

Stamford-based SpringWorks Therapeutics, a commercial-stage biotech company developing treatments for rare diseases and ...

Market OverviewThe Orphan Drugs Market is a specialized sector within the pharmaceutical industry that focuses on the ...

GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN –– Approval based on positive data ...

Roche’s Evrysdi tablet receives US FDA approval for the treatment of spinal muscular atrophy: Basel Friday, February 14, 2025, 13:00 Hrs [IST] Roche, announced that the US Food ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

Ariceum Therapeutics (Ariceum), a private biotech company developing radiopharmaceutical products for the diagnosis and treatment of certain hard-to-treat cancers, today announced that the U.S. Food ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...