Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study

Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners. The post Ionis Pharma’s Drug for Rare Disease With No Approved Therapies Meets Goals of Pivotal Study appeared…

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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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Watch: How Controversies Over Vaccine Changes Affect You

Céline Gounder, KFF Health News’ editor-at-large for public health, appeared on CBS News’ “Face the Nation With Margaret Brennan” on Aug. 31 to discuss leadership changes at the Centers for Disease Control and Prevention and vaccine policy concerns. Gounder noted that the departures of top officials and confusion over how to get vaccines affect the…

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Health Insurance Marketplace Calculator Embed Instructions

Usage Guidelines KFF encourages other organizations to embed the Health Insurance Marketplace Calculator on their websites. To do so, follow the instructions below. Please do not alter the embed code in any way without advanced permission.The calculator widget is intended to be embedded in areas between 300 px and 800 px wide and will expand to…More

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