After asset purchase from Travere, Mirum's Ctexli crosses FDA finish line in rare metabolic disorder
The approval makes chenodiol—already cleared for decades to treat radiolucent gallstones under the brand name Chenodal—the ...
The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in ...
The FDA recently granted EXG-34217 Rare Pediatric Disease Designation (RPDD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.
Market OverviewThe Orphan Drugs Market is a specialized sector within the pharmaceutical industry that focuses on the ...
MeiraGTx stock rose after publishing promising results from the first human trial using gene therapy to treat ...
“This designation, following the Orphan Drug designation granted last month, reinforces the promise of amezalpat as a ...
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News Medical on MSNTampere University licenses to Theravia a drug molecule intended for rare and severe skin disordersTampere University has licensed a drug molecule intended for treating Epidermolysis Bullosa (EB), a group of rare and severe ...
Ensoma Inc.’s lead program, EN-374, has been granted orphan drug and rare pediatric disease designations by the FDA for the ...
HOUSTON, TX / ACCESS Newswire / February 18, 2025 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP)("CNS" or the "Company"), a ...
Chimerix holds the potential to have the first approval of dordaviprone for treating recurrent H3 K27M mutant high-grade ...
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