BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network's 10th Annual WORLDSymposium(TM) 12 Abstracts on MPS diseases and 3 on Pompe Disease 10-Year Naglazyme® (galsulfase) Data Suggests Increased Survival of MPS VI Patients SAN RAFAEL, Calif., Feb. 13, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today 15 data presentations at the Lysosomal Disease Network's 10^th Annual WORLDSymposium™ from February 11-13 in San Diego, California. "Since our founding 16 years ago, BioMarin has been deeply committed to patients suffering from lysosomal storage disorders and continues to build on our knowledge of MPS diseases, enzyme replacement therapies and technologies to deliver those therapies to a specific place in the cell," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin."The depth and breadth of our research covers exciting new molecules that have the potential to change the course of these difficult to treat diseases.We are so grateful to the patients who participate in clinical trials, and they motivate us to bring the hope of new treatment options." Paul Harmatz, M.D., Associate in Gastroenterology and Nutrition at the Children's Hospital and Research Center inOakland, California will present data tracking MucopolysaccharidosisType VI (MPS VI or Maroteaux-Lamy syndrome) patients over a 10 year period who had been on treatment with Naglazyme® (galsulfase).The data suggests that patients treated with Naglazyme lived longer, had long-term improvement in endurance, and experienced improvement in pulmonary function and growth. "The 10-year data on galsulfase provides evidence that reinforces the accepted thinking that early intervention with an enzyme replacement can make a big difference to patients," said Paul Harmatz, M.D."As a treating physician, therapies that can change the long-term outcome of disease are the gold standard." Presentations: MPS VI (Maroteaux-Lamy syndrome) Title Authors Galsulfase for Mucopolysaccharidosis Type VI: Harmatz P, Hendriksz CJ, Analysis of Clinical Data Since 2000 Giugliani R, Braunlin E, Quartel A PRESENTATION Growth Charts for Individuals with Quartel A, Graham S, Harmatz Mucopolysaccharidosis VI (Maroteaux-Lamy P, Lin P Syndrome) MPS IVA (Morquio A syndrome) Title Authors Burden of Disease Suffered by Patients with Hendriksz CJ, Lavery C, Coker M, Morquio A Syndrome: Results from Ucar S, Jain M, Bell L, Lampe C Patient-Reported Outcomes Survey The Heart and Cardiovascular System in Kampmann C, Lampe C,Reinke J, Patients with Morquio A Syndrome Mengel E,Gökce S,Beck M, Kuroczynski W Burden of Disease Suffered by Caregivers of Lampe C, Hendriksz CJ, Lavery C, Patients with Morquio A Syndrome: Results of Coker M, Ucar M, Bell L, Jain M a Self-Reported Outcomes Survey Prediction of the Molecular Consequences of Kubaski F, Brusius-Facchin AC, Amino Acid Substitutions in the GALNS Gene Nemetz-Bochernitsan A, Giugliani using In Silico Tools R, Leistner-Segal S PRESENTATION Keratan Sulfate (KS) Analysis in Morquio A Patients: LC-MS/MS Analysis of KS Miller N, Taniguchi G, Decker C, Disaccharides Demonstrates that Urine is a ZhouH, Shediac R, Quartel A Better Source than Plasma to Monitor Dynamic Change in KS Morquio A Locus-Specific Database: A Frameworkfor a Curated Database to Identify Ryles A, Du C, Oron T, Atwood R, and Characterize Pathogenic Variants in the Mooney SD, Francis-Lyon P GALNS Gene MPS IIIB (Sanfilippo syndrome) Title Authors Engineering of a Recombinant NAGLU Fusion Aoyagi-Scharber M, Christianson Protein with Insulin‐Like Growth Factor 2 T, Wendt DJ, Pascale MNT, Yip Leads to Improved Cellular Uptake via a BK,Holtzinger J, Chen Z, Glycosylation‐Independent Lysosomal Targeting Woloszynek J, Cheung DS, Lo MJ, Pathway Dickson P, Fitzpatrick PA, LeBowitz JH Intraventricular Enzyme Replacement Therapy Kan SH, Le S, Vincelette J, with Glycosylation-Independent Lysosomal Bullens S, Brown J, Ohmi K, Targeted NAGLU in the Brain of Sanfilippo B Lotshaw E, Aoyagi-Scharber M, Mice Crawford B, Bunting S, Neufeld E, Dickson P PRESENTATION MPS General Title Authors Target-Population Screening for Lysosomal Storage Cobos PN, Santer R, Zoltan Disorders - A Highly Efficient Tool for the L Diagnosis of Patients The Development and Validation of Dried Blood Spot Ullal AJ, Millington DS, Enzymatic Assays for MPS Type IVA (Morquio) and WoodTC, Bali D Type VI (Maroteaux-Lamy) Syndromes Pompe disease Title Authors Preliminary Clinical Efficacy and Byrne B, Barhon R, Barshop B, Safety of BMN 701, GILT-tagged Bratkovic D, Desnuelle C, Geberhiwot Recombinant Human Acid Alpha T, Henderson R, Hughes D, Laforet P, Glucosidase (rhGAA), in Late Onset Mengel E, Roberts M, Chinnapolamada G, Pompe Disease: Results of an Extension Schweighardt B, Tompkins T, Lang W, Study LeBowitz J PRESENTATION BMN 701 Mediated Receptor LeBowitz J, Maga J, Schooler B, Chen Redistribution is Responsible for G, Pungor E, Prince B, Liu G, Xia Y Increased Uptake A Comparison of Pharmacological Activity of Multiple Production Lots of Peng J, Cahayag R, Crockett L, Fox M, BMN 701 by Glycogen Clearance in a O'Neill CA, LeBowitz J, Tsuruda L Mouse Model of Pompe Disease About BioMarin BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for MPS VI, a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include VIMIZIM™ (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase 3 clinical development for the treatment of MPS IVA, PEG PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 1/2 clinical development for the treatment of Pompe disease, BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease, which is currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the treatment of hemophilia A and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release. Vimizim™ is our trademark, and BioMarin^®, Naglazyme^®, Kuvan^®, Firdapse^® are registered trademarks of BioMarin Pharmaceutical Inc. Aldurazyme^® is a registered trademark of BioMarin/Genzyme LLC. CONTACT: Investors: Traci McCarty BioMarin Pharmaceutical Inc. (415) 455-7558 Media: Debra Charlesworth BioMarin Pharmaceutical Inc. (415) 455-7451 BioMarin Pharmaceutical Inc. Logo
BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network's 10th Annual WORLDSymposium
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