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BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.
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New Investigator-Led Study Shows VOXZOGO Significantly Increased Bone Length While Maintaining Bone Strength Through 5 Years of Observation in Children with Achondroplasia Phase 2 and 3 Data on VOXZOG...
Data from Longest and Largest Hemophilia Gene Therapy Study Show Durable and Sustained Bleed Control and Factor VIII Expression Maintained Four Years Post-ROCTAVIAN Infusion Additional Data Show Meani...
Wall Street regards the healthcare sector as a constant source of innovation, while biotechnology stands at the pinnacle of groundbreaking medical advancements. The long-term potential of robust biote...
Beacon Pointe Advisors LLC lessened its stake in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 0.7% during the fourth quarter, according to the company in its most recent...
We live in the best of times where life is not capped at 45 or 50 years of age, all thanks to wonderful discoveries in the field of biotechnology. The resources being invested to produce new drugs and...
SAN RAFAEL, Calif. , May 8, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that Alexander Hardy, President, and Chief Executive Officer of BioMarin will present at th...
New drug approvals and pipeline progress are likely to help maintain the momentum for the Zacks Biomedical and Genetics industry. A strong portfolio and solid pipeline progress position BMRN, BPMC, IM...
Investigator-Sponsored Phase 2 Study Demonstrated Positive Results in Children with Noonan Syndrome, Idiopathic Short Stature and Other Growth-Related Conditions BioMarin-Sponsored Phase 3 Data Show...
Amid the current earnings season, with brokers actively revising price targets, Citigroup (NYSE: C ) recently reduced its 12-month targets for several companies. Therefore, we discuss three stocks aff...