1+ mon, 1+ week ago  (248+ words) James Treat, MD, Professor of Clinical Pediatrics and Dermatology at the Children's Hospital of Philadelphia to present clinical results on Wednesday, May 20, 2026, at 4: 30 p. m. ET WAYNE, Pa. , April. .. Palvella Therapeutics to Present QTORIN" Rapamycin Results from Phase 3 SELVA and Phase 2 TOIVA…...

4+ week, 1+ day ago  (247+ words) Intellia Therapeutics" lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to the swelling attacks from the rare disease hereditary angioedema. Intellia has filed a regulatory submission for this genetic medicine, which…...

1+ mon, 1+ hour ago  (449+ words) Intellia Therapeutics said its Crispr-based treatment for a rare swelling condition met its goals in a late-stage trial, marking a milestone for the field of gene editing and putting the company on track to seek approval from the U. S. Food and…...

1+ day, 14+ hour ago  (563+ words) The deaths of 20 patients in Japan have prompted scrutiny of a drug produced by Thousand Oaks-based Amgen. Sales of the problematic Amgen Inc. drug Tavneos resumed late last week in Japan with a new warning label after several patient deaths…...

3+ week, 5+ day ago  (231+ words) STAT is a media company focused on finding and telling compelling stories about health, medicine, and scientific discovery. We produce daily news, investigative articles, and narrative projects in addition to multimedia features, podcasts, and opinion pieces. We tell our stories…...

6+ day, 4+ hour ago  (167+ words) Salt Lake City-based infrastructure company addresses the architectural failure underneath three decades of population-averaged precision medicine, with founder and Business Development team available for meetings June 22-25 at the Bio Utah pavilion "Adverse drug reactions cost the American healthcare system thirty…...

6+ hour, 56+ min ago  (490+ words) A longstanding mystery in Parkinson's disease research has been why some individuals carrying pathogenic variants that increase their risk of PD go on to develop the disease, while others who also carry such variants do not. The prevailing theory has…...

1+ hour, 56+ min ago  (160+ words) Utilizing a novel dual-strike mechanism, the proprietary combination agent has the potential to deliver a low pill burden option to manage existing and future outbreaks Interactions planned with the US Centers for Disease Control and Prevention (CDC), and ongoing with the…...

2+ day, 17+ hour ago  (372+ words) Gov. Phil Scott speaks at a press conference on Thursday, April 23. MONTPELIER " The National Organization for Rare Disorders celebrated the signing of House Bill 46 into law by Governor Phil Scott, establishing a Rare Disease Advisory Council (RDAC) in Vermont. This…...

1+ day, 2+ hour ago  (81+ words) Brain Cancer Canada (BCC), Canada's only 100% volunteer-driven charity dedicated exclusively to funding brain cancer research nationwide is proud to award $50, 000 to an exciting research project led. .. About Brain Cancer Canada As a fully volunteer-based organization, Brain Cancer Canada ensures that…...