WebFeb 28, 2024 · In this open-label gene therapy study, infusions for MPS type VI occurred without severe adverse events. In the high-dose cohort, serum active arylsulfatase B reached 30% to 100% of normal. A modest urinary GAG increase did not require reintroduction of enzyme replacement therapy. Clinical deterioration was not noted for up... Webn engl j med 377;23 nejm.orgDecember 7, 2024 2217 Hemophilia B Gene Therapy with Factor IX Variant first author prepared the first draft of the manu - script and, with two authors who are employees of the sponsor, wrote the manuscript with criti-cal input from all the coauthors and without editorial assistance. The authors assume respon -
Matthew T.A. Krusen on LinkedIn: In Gratitude for mRNA Vaccines NEJM
WebIn the high-dose cohort, the median annualized bleeding rate decreased from 16 events before the study to 1 event after gene transfer, and factor VIII use for bleeding ceased in … WebGrateful for the support and for the opportunity to talk about the Patient journey during the development of complex cell and gene therapies. We continue to… Dr. Panteli Theocharous على LinkedIn: Delivering on the promise of … brushed gold toilet bowl brush
Genetics General NEJM Evidence
WebPHILADELPHIA, Dec. 12, 2024 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated gene therapy company today announced updated multi-year results from its Phase 1/2 clinical trial of investigational SPK-8011 for patients with hemophilia A. With up to five … WebAug 19, 2024 · Emerging gene therapy and gene-editing technologies will have a growing impact on patient lives and health care delivery. The authors analyzed a decade of data … WebSep 16, 2024 · In the last decade, enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered adeno-associated virus (AAV)-based liver-directed gene therapy in patients with severe hemophilia B were reported in 2011, many gene therapy studies have been … brushed gold toilet brush