Phase 3 Randomized Trial Results of DTX401 AAV Gene Therapy for the Treatment of GSDIa.

Address: Montreal Children's Hospital, Montreal, Quebec, Canada.; Rady Children's Health, Orange, California, USA.; University of Groningen, University Medical Center Groningen, Beatrix Children's Hospital, Section of Metabolic Diseases, Groningen, the Netherlands.; UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands.; Mount Sinai, New York, New York, USA.; Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBERER, Santiago de Compostela, Spain.; Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.; Duke University Medical Center, Durham, North Carolina, USA.; University of Connecticut, Farmington, Connecticut, USA.; University of Utah, Salt Lake City, Utah, USA.; Centre for Inherited Metabolic Diseases, Rigshospitalet, Copenhagen, Denmark.; Hospital Clínico Universitario de Santiago de Compostela, IDIS, CIBEROBN, Santiago de Compostela, Spain.; Children's Hospital Colorado, Aurora, Colorado, USA.; Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil.; University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Cleveland Clinic Childrens, Cleveland, Ohio, USA.; Department of Pediatrics, Division of Medical Genetics, McGovern Medical School at the University of Texas Health Science Center at Houston (UTHealth Houston) and Children's Memorial Hermann Hospital, Houston, Texas, USA.; Pediatric Clinic and Endocrinology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy.; Department of Translational Medicine, University of Naples Federico II, Naples, Italy.; Department of Endocrinology, University of Groningen, University Medical Center Groningen, Groningen, the Netherlands.; Boston Children's Hospital, Boston, Massachusetts, USA.; Ultragenyx Pharmaceutical Inc., Novato, California, USA.