BioMarin Leadership and External Medical
Experts to Discuss 7 Product Candidates in Clinical
Development as well as ROCTAVIAN and VOXZOGO New Indication
Opportunities
R&D Day to be Webcast Beginning at
8:00 a.m. Eastern Time September 12th
SAN
RAFAEL, Calif., Sept. 6,
2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
(NASDAQ: BMRN), a global biotechnology company dedicated to
transforming lives through genetic discovery, will host an
R&D Day at 8:00 a.m. ET on Tuesday,
September 12, 2023. BioMarin management and external experts
will provide an update to the investment community on the company's
development portfolio.
Key topics to be discussed at BioMarin's R&D Day:
Pipeline Programs:
- BMN 255 for hyperoxaluria in nonalcoholic fatty liver
disease
- BMN 349 for alpha-1 antitrypsin deficiency
- BMN 331 for hereditary angioedema
- BMN 351 for Duchenne muscular dystrophy
- BMN 293 for myosin binding protein C3 hypertrophic
cardiomyopathy
- New Candidate BMN 365 for plakophilin-2 (PKP2)
arrhythmogenic cardiomyopathy
- New Candidate BMN 355 for long-QT syndrome types 2 and
3
ROCTAVIAN panel discussion on severe hemophilia A and new
indications:
- Professor 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, Johannesburg
- Professor Amit Nathwani, MBChB,
FRCP, RCPath, Ph.D.- Director of the Katharine Dormandy Hemophilia
Centre, Royal Free Hospital, London
- Guy 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
Angeles
VOXZOGO panel discussion on achondroplasia and new
indications:
- Andrew 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 Trust, Evelina Children's Hospital, London, UK
- Bradley Miller, M.D., Ph.D. -
Director, Division of Endocrinology, M Health Fairview Masonic
Children's Hospital; Professor, Department of Pediatrics,
University of Minnesota Medical School,
Minneapolis, Minnesota
Live Audio Webcast
An audio webcast of the presentations will be available live.
You can access the webcasts at: https://investors.biomarin.com/. An
archived version of the remarks will also be available through the
Company's website for a limited time following the event.
About BioMarin
Founded in 1997, BioMarin is a global biotechnology company
dedicated to transforming lives through genetic discovery. The
company develops and commercializes targeted therapies that address
the root cause of the genetic conditions. BioMarin's unparalleled
research and development capabilities have resulted in eight
transformational commercial therapies for patients with rare
genetic disorders. The company's distinctive approach to drug
discovery has produced a diverse pipeline of commercial, clinical,
and pre-clinical candidates that address a significant unmet
medical need, have well-understood biology, and provide an
opportunity to be first-to-market or offer a substantial benefit
over existing treatment options. For additional information, please
visit www.biomarin.com.
Contacts:
|
Investors
|
Media
|
Traci
McCarty
|
Andrew Villani
|
BioMarin
Pharmaceutical Inc.
|
BioMarin Pharmaceutical Inc.
|
(415)
455-7558
|
(628) 269-7393
|
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SOURCE BioMarin Pharmaceutical Inc.