Applied Therapeutics Drug Is Denied FDA Approval in Rare Inherited Metabolic Disease

The FDA cited deficiencies in its govorestat’s application in galactosemia, a rare metabolic disease, Applied Therapeutics said. The biotech is still on track to submit a separate application for the drug in sorbitol dehydrogenase (SORD) deficiency, another rare disease that has no FDA-approved therapies. The post Applied Therapeutics Drug Is Denied FDA Approval in Rare…

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Quantifying the Altruism Value for a Rare Pediatric Disease: Duchenne Muscular Dystrophy

That is the title of a paper recently published in AJMC with co-authors Suhail Thahir, Alexa Klimchak, Ivana Audhya, Lauren Sedita, and John A. Romley. The abstract is below. Objectives: To quantify the magnitude of altruism value as applied to a hypothetical new treatment for a rare, severe pediatric disease: Duchenne muscular dystrophy (DMD). Study Design: Prospective…

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Will AI help patients appeal insurance denials?

It looks like the answer is ‘yes’. KFF reports that Holden Karau, a software engineer from the San Francisco Bay Area, created an AI tool to help appeal insurance denials. She calls it: Fight Health Insurance,  How does it work? You’ve gotta make a scan of the denial letter from your insurance company, and run…

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Beyond the Hype: The Scientific and Commercial Future of Microbiome Medicine

By aligning innovative scientific insights with strategic commercial pathways, the industry has the potential to fulfill its early promise – delivering safe, effective therapies that improve patient outcomes and create new opportunities for growth in the biopharmaceutical sector. The post Beyond the Hype: The Scientific and Commercial Future of Microbiome Medicine appeared first on MedCity…

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