OV101 program in Angelman syndrome to pause pending full analysis of NEPTUNE trial and discussions with FDA Pivotal studies of OV935 (soticlestat) in Dravet syndrome and Lennox-Gastaut syndrome ...
MIDDLETON, Mass., Sept. 22, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...
For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman syndrome. James was just a toddler when he was diagnosed with the rare ...
SARASOTA, Fla. and NOVATO, Calif., May 04, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development ...
Ultragenyx (RARE) Pharmaceutical announced that the first patient has been dosed in the pivotal Phase 3 Aspire study, NCT06617429, evaluating the efficacy and safety of GTX-102, its investigational ...
The Pinball Lounge at 78th Street Sudios offers an ideal setting for this unique fundraiser. The event runs from 4-6 pm, providing families with an accessible Sunday afternoon activity. True to its ...
Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 NOVATO, Calif., ...
NOVATO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE) today announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to ...
ORLANDO, Fla.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that ...
A rare genetic condition leads to developmental difficulties, happy disposition. Oct. 22, 2008— -- Six-year-old Ryan Ravellette is a happy and sociable child; he has a smile that can light up a ...
It wasn’t long after our son Theodore was born that my husband, Daniel, and I noticed how incredibly happy he was. As in “Clap along if you feel like a room without a roof” kind of happy. He barely ...
Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 “The accelerated ...
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