Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys

Sarepta Therapeutics said the Duchenne muscular dystrophy patient recently had a cytomegalovirus infection, which can damage the liver and may have contributed to the fatality. It’s the first patient death out of more than 800 patients treated with Elevidys, a gene therapy for the rare muscle-wasting disease. The post Liver Failure-Associated Death Reported in Patient…

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Workplace Mental Health at Risk as Key Federal Agency Faces Cuts

In Connecticut, construction workers in the Local 478 union who complete addiction treatment are connected with a recovery coach who checks in daily, attends recovery meetings with them, and helps them navigate the return to work for a year. In Pennsylvania, doctors applying for credentials at Geisinger hospitals are not required to answer intrusive questions…

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BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition

BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers. The post BioMarin’s Presence in Rare Enzyme Disorders Grows With $4.8B Amicus Therapeutics Acquisition appeared first on MedCity News.

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KFF Health News’ ‘What the Health?’: On Capitol Hill, RFK Defends Firings at CDC

The Host Julie Rovner KFF Health News @jrovner @julierovner.bsky.social Read Julie’s stories. Julie Rovner is chief Washington correspondent and host of KFF Health News’ weekly health policy news podcast, “What the Health?” A noted expert on health policy issues, Julie is the author of the critically praised reference book “Health Care Politics and Policy A…

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Would delinking PBM reimbursement from rebates save money?

According to a Health Affairs Scholar paper by Geoffrey Joyce out this month, the answer is ‘yes’. He writes: Most PBM contracts tie their compensation to a percentage of a drug’s list price, creating a financial incentive to favor high-cost, high-rebate drugs on plan formularies at the expense of lower-cost generics and biosimilars. Furthermore, the…

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