BioMarin Presents 15 Abstracts From Basic Research to Clinical Trials at Lysosomal Disease Network's 10th Annual WORLDSymposium

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

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