Neopharmed Gentili is acquiring European rights to Orladeyo, bringing the Italy-based pharmaceutical company into rare disease. BioCryst’s Orladeyo is approved for treating patients with the rare disease hereditary angioedema. The post BioCryst Pharma Sells European Business for Rare Disease Drug in $250M Deal appeared first on MedCity News.
Check out my new paper titled “Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies“. The abstract is below. Purpose: Traditional value assessment frameworks are challenged in comprehensively assessing the societal value new therapies bring to individuals with rare, progressive, genetic, fatal, neuromuscular diseases such as Duchenne muscular dystrophy (DMD). The…
Amid the challenges of adulthood, one rite of passage is unique to the United States: the need to find your own health insurance by the time you turn 26. That is the age at which the Affordable Care Act declares that young adults generally must get off their family’s plan and figure out their coverage…
This issue brief describes the hardship exception for individuals living in counties with high unemployment, and using the most recent available county-level unemployment data, estimates the number of counties that could meet the criteria for this exception and the number of expansion enrollees living in those counties who could be exempt from work requirements.
For nursing to shape its future in an AI-driven world, its leaders and practitioners must be present, informed, and engaged in every step of its development and deployment. The post The Critical Need For Nurses To Play An Active Role In AI Implementation appeared first on MedCity News.
Shirley Ryan AbilityLab Earns New Joint Commission Sustainability Award With support from Compass One Healthcare, Shirley Ryan AbilityLab has reached another milestone in its commitment to environmental stewardship. The Chicago-based rehabilitation hospital, renowned for treating patients with stroke, spinal cord, and brain injuries, has earned The Joint Commission’s new Sustainability Healthcare Certification. It is the…
Zevra Therapeutics drug Miplyffa is now FDA approved for Niemann-Pick disease type C, a lysosomal storage disorder. The affirmative regulatory decision took into account additional clinical data generated since the FDA turned down a submission for the drug in 2021. The post FDA Approval Makes a Zevra Drug the First Therapy for Rare and Fatal…
Ohio Republican Sen. JD Vance and Minnesota Democratic Gov. Tim Walz met in an Oct. 1 vice presidential debate hosted by CBS News that was cordial and heavy on policy discussion — a striking change from the Sept. 10 debate between Vice President Kamala Harris and former President Donald Trump. Vance and Walz acknowledged occasional agreement on…
GLP-1 drugs dominate the obesity market, but Aardvark Therapeutics aims to curb appetite by going after a different target: TAS2 receptors in the gut. The biotech’s lead drug candidate is in Phase 3 testing in Prader-Willi syndrome, a genetic disorder that causes persistent hunger leading to overeating. The post Aardvark Therapeutics’ Pursuit of New Obesity…