Stay updated with breaking news from Donaldb Kohn. Get real-time updates on events, politics, business, and more. Visit us for reliable news and exclusive interviews.
Advanced RP-L102 for Fanconi Anemia towards regulatory reviews; EMA accepted MAA for review and BLA submission anticipated in the first half of 2024 Continued preparations to launch LV portfolio...
For many patients, pushing for access to unproven treatments is their best chance of survival. And that’s worth the risk.
For the readers interested in the stock health of Rocket Pharmaceuticals Inc. (RCKT). It is currently valued at $17.23. When the transactions were called off in the previous session, Stock hit the highs of $17.575, after setting-off with the price of $17.33. Company’s stock value dipped to $16.54
— Continued to advance clinical gene therapy programs for the treatment of Danon Disease, Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) —— Updates on all four programs to be presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGC...
email article A novel gene therapy for severe combined immunodeficiency from adenosine deaminase deficiency (ADA-SCID) restored immune cell counts without death or need for enzyme replacement therapy, researchers reported. Survival without needing to re-initiate pegylated ADA enzyme replacement therapy, or have a rescue allogeneic hematopoietic stem cell transplantation (AHCT), was 95% to 97% at last follow-up at 24 or 36 months across three studies pooled together by Donald B. Kohn, MD, of the University of California Los Angeles, and colleagues. Those rates compare favorably with the ...