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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Make Us Swoon: Send In Your Health Policy Valentines

Affordable health care is our love language. Stoke the fire by submitting your sweetest “Health Policy Valentines.” We want to see your clever, heartfelt, or hilarious tributes to the policies that shape health care. An esteemed panel of editors will review entries. We’ll share favorites on our social media channels, and our staff will pick…

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LLM in HEOR: An evaluation framework

Health economics and outcomes research has already started to use AI tools such as large language models (LLM) across a diverse set of study types, including systematic literature reviews (SLR), health economic modelling (HEM) and real-world evidence (RWE). In SLRs, LLMs can assist with abstract and full-text screening, bias assessment, data extraction, and automating meta-analysis…

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The Same Processes that Make Smartphones a Risk for Young People Must Be Harnessed to Provide Beneficial Digital Medical Treatments For All

Addressing difficult-to-treat diseases and disorders, quickly, safely, and with little to no side effects is a positive boon to our overburdened health system. The post The Same Processes that Make Smartphones a Risk for Young People Must Be Harnessed to Provide Beneficial Digital Medical Treatments For All appeared first on MedCity News.

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