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 ...
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Hosted on MSNThe New Gold Rush in Rare Disease Treatments and Merck KGaA’s Acquisition of SpringWorks TherapeuticsFor many years, the pharmaceutical business concentrated mostly on blockbuster medications, which are cures for common ...
The FDA recently granted EXG-34217 Rare Pediatric Disease Designation (RPDD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.
The FDA has granted orphan drug designation to the oral agent OPN-6602 for the treatment of patients with relapsed or ...
The FDA has approved Ctexli for the treatment of cerebrotendinous xanthomatosis (CTX), a rare lipid storage disease, in ...
MeiraGTx (MGTX) stock rose 7% after publishing promising results from the first human trial using gene therapy to treat AIPL1-associated severe retinal dystrophy in children. Across all trials so far, ...
Market OverviewThe Orphan Drugs Market is a specialized sector within the pharmaceutical industry that focuses on the ...
“Educating people about the signs and symptoms of MCT8 deficiency, as well as the journey of patients like my son Colton is critically important,” added Favre. “On Rare Disease Day and every day, MCT8 ...
Chimerix holds the potential to have the first approval of dordaviprone for treating recurrent H3 K27M mutant high-grade ...
Cardiol Therapeutics' CardiolRx shows promise in treating heart conditions. Click here to read more about CRDL stock and why ...
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