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BioMarin to Present Innovative Pipeline at R&D Day on September 12th in New York
Live Audio Webcast to Begin at 8:00 a.m. Eastern Time SAN RAFAEL, Calif., Sept. 11, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Live Audio Webcast to Begin at 8:00 a.m. Eastern Time \nSAN RAFAEL, Calif., Sept. 11, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will update the investor community on the company's research and development portfolio, including early-stage product candidates and new indication opportunities with VOXZOGO and ROCTAVIAN. In addition, external clinicians from leading global institutions will participate in panel discussions.\n\n \n \n \n \n \n \n\n \n\"We look forward to sharing progress across our earlier-stage, next generation product candidates from the most diverse and high-potential pipeline in our company's history,\" said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin.\nPipeline programs to be presented:\nTherapeutic Focus\nCandidate\nTechnology\nCondition\nMusculoskeletal/ Metabolic\nBMN 255\nSmall molecule\nHyperoxaluria in nonalcoholic fatty liver disease\nBMN 351\nExon 51 oligonucleotide \nDuchenne muscular dystrophy \nCardiovascular\nBMN 293\nAAV gene therapy\nMyosin binding protein C3 hypertrophic cardiomyopathy\nBMN 365 \nAAV gene therapy\nPlakophilin-2 (PKP2) arrhythmogenic cardiomyopathy\nBMN 355 \nMonoclonal antibody\nLong QT syndrome types 2 and 3\nOther\nBMN 349\nSmall molecule\nAlpha-1 antitrypsin deficiency \nBMN 331\nAAV gene therapy \nHereditary angioedema\nROCTAVIAN panel discussion on severe hemophilia A and new indications: \nProfessor Johnny Mahlangu, MBBCh, MMed, FCPath – Director Haemophilia Comprehensive Care, Charlotte Maxeke Johannesburg Academic Hospital, Professor of Haematology, University of the Witwatersrand and National Health Laboratory Service, JohannesburgProfessor Amit Nathwani, MBChB, FRCP, RCPath, Ph.D.- Director of the Katharine Dormandy Hemophilia Centre, Royal Free Hospital, LondonGuy Young, M.D. – Director, Hemostasis and Thrombosis Center and Clinical Coagulation Laboratory, Children's Hospital; Professor of Pediatrics, University of Southern California's Keck School of Medicine, Los AngelesVOXZOGO panel discussion on achondroplasia and new indications: \nAndrew Dauber, M.D. - Chief of Endocrinology, Children's National Hospital, Washington, D.C.Melita Irving, M.B.B.S. - Consultant in Clinical Genetics, Guy's and St. Thomas' NHS Foundation Trus...