Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and ...
Every Cure was co-founded by a Penn immunologist after discovering an off-label treatment for his own rare disease.
The Avestagenome Project® International Pvt. Ltd. (AGENOME) & Avesthagen Limited, in partnership with the Tata Institute for ...
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...
Percheron Therapeutics Ltd has submitted a non-binding proposal to an international pharmaceutical company to in-license a new drug development program focused on a rare neurological disease.
We recently published a list of the 10 Best Performing Pharma Stocks So Far in 2025. In this article, we are going to take a ...
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 ...
EQS-News: Benzinga / Key word (s): Science Soligenix's Leadership Aims To Drive Growth In Rare Disease Markets In 2025 And ...
Years of translational research have successfully improved treatment outcomes for common dermatological conditions such as ...
BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...
Under terms of the deal, Stoke will spearhead global development of zorevunersen, while Biogen will receive exclusive rights ...
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The 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 ...
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