FDA-approved AVLAYAH, BBB TransportVehicle platform, $1.17B liquidity & runway—assess valuation, pipeline upside now.
After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination ...
The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for tividenofusp alfa for the treatment of Hunter syndrome, also known as ...
Ever since her 15-year-old son Cole was diagnosed with a rare, progressive illness called Hunter syndrome when he was 2, Kim Stephens has been waiting. Waiting to see if Cole will lose his ability to ...
Denali Therapeutics, Inc. DNLI announced that the FDA has accepted for review its biologics license application (BLA) for lead-pipeline candidate tividenofusp alfa. The BLA is seeking accelerated ...
Grant is an M.D./M.B.A. student at Harvard Medical School and Harvard Business School, a researcher at Boston Children’s Hospital, a board member of Project Alive, and the president and founder of ...
New Berlin boy with a rare genetic disease receives his first dose of a newly approved FDA treatment
A 6-year-old New Berlin boy with Hunter syndrome received his first dose of a newly FDA-approved medication, giving his ...
Jeanette Henriquez, on the left, stands with her two children, Jasmine Henriquez and Dominic Henriquez. Dominic Henriquez was diagnosed with Hunter Syndrome in 2011 before the condition was added to ...
REGENXBIO specializes in gene therapies using proven AAV technology. RGX-121, targets Hunter syndrome and is under FDA review, with a PDUFA date set for November 9, 2025. We believe the upcoming FDA ...
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