This Day On The Street
Continue to site
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

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 in Oakland, California will present data tracking Mucopolysaccharidosis Type 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."

MPS VI (Maroteaux-Lamy syndrome)  
Title Authors
Galsulfase for Mucopolysaccharidosis Type VI: Analysis of Clinical Data Since 2000 Harmatz P, Hendriksz CJ, Giugliani R, Braunlin E, Quartel A
Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome) Quartel A, Graham S, Harmatz P, Lin P
MPS IVA (Morquio A syndrome)  
Title Authors
Burden of Disease Suffered by Patients with Morquio A Syndrome: Results from Patient-Reported Outcomes Survey Hendriksz CJ, Lavery C, Coker M, Ucar S, Jain M, Bell L, Lampe C
The Heart and Cardiovascular System in Patients with Morquio A Syndrome   Kampmann C, Lampe C, Reinke J, Mengel E, Gökce S, Beck M, Kuroczynski W
Burden of Disease Suffered by Caregivers of Patients with Morquio A Syndrome: Results of a Self-Reported Outcomes Survey Lampe C, Hendriksz CJ, Lavery C, Coker M, Ucar M, Bell L, Jain M
Prediction of the Molecular Consequences of Amino Acid Substitutions in the GALNS Gene using In Silico Tools   Kubaski F, Brusius-Facchin AC, Nemetz-Bochernitsan A, Giugliani R, Leistner-Segal S
Keratan Sulfate (KS) Analysis in Morquio A Patients: LC-MS/MS Analysis of KS Disaccharides Demonstrates that Urine is a Better Source than Plasma to Monitor Dynamic Change in KS Miller N, Taniguchi G, Decker C, Zhou H, Shediac R, Quartel A
Morquio A Locus-Specific Database: A Framework for a Curated Database to Identify and Characterize Pathogenic Variants in the GALNS Gene Ryles A, Du C, Oron T, Atwood R, Mooney SD, Francis-Lyon P
MPS IIIB (Sanfilippo syndrome)  
Title Authors
Engineering of a Recombinant NAGLU Fusion Protein with Insulin‐Like Growth Factor 2 Leads to Improved Cellular Uptake via a Glycosylation‐Independent Lysosomal Targeting Pathway Aoyagi-Scharber M, Christianson T, Wendt DJ, Pascale MNT, Yip BK, Holtzinger J, Chen Z, Woloszynek J, Cheung DS, Lo MJ, Dickson P, Fitzpatrick PA, LeBowitz JH
Intraventricular Enzyme Replacement Therapy with Glycosylation-Independent Lysosomal Targeted NAGLU in the Brain of Sanfilippo B Mice Kan SH, Le S, Vincelette J, Bullens S, Brown J, Ohmi K, Lotshaw E, Aoyagi-Scharber M, Crawford B, Bunting S, Neufeld E, Dickson P
MPS General  
Title Authors
Target-Population Screening for Lysosomal Storage Disorders - A Highly Efficient Tool for the Diagnosis of Patients Cobos PN, Santer R, Zoltan L
The Development and Validation of Dried Blood Spot Enzymatic Assays for MPS Type IVA (Morquio) and Type VI (Maroteaux-Lamy) Syndromes Ullal AJ, Millington DS, Wood TC, Bali D
Pompe disease  
Title Authors
Preliminary Clinical Efficacy and Safety of BMN 701, GILT-tagged Recombinant Human Acid Alpha Glucosidase (rhGAA), in Late Onset Pompe Disease: Results of an Extension Study Byrne B, Barhon R, Barshop B, Bratkovic D, Desnuelle C, Geberhiwot T, Henderson R, Hughes D, Laforet P, Mengel E, Roberts M, Chinnapolamada G, Schweighardt B, Tompkins T, Lang W, LeBowitz J
BMN 701 Mediated Receptor Redistribution is Responsible for Increased Uptake LeBowitz J, Maga J, Schooler B, Chen G, Pungor E, Prince B, Liu G, Xia Y
A Comparison of Pharmacological Activity of Multiple Production Lots of BMN 701 by Glycogen Clearance in a Mouse Model of Pompe Disease Peng J, Cahayag R, Crockett L, Fox M, O'Neill CA, LeBowitz J, Tsuruda L

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.

1 of 2

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

David Peltier identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free


Chart of I:DJI
DOW 17,798.49 -14.90 -0.08%
S&P 500 2,090.11 +1.24 0.06%
NASDAQ 5,127.5250 +11.3820 0.22%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs