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BioMarin Announces 14 Presentations at 15th Annual WORLDSymposium™ 2019
Presentations to Highlight Results from Studies for the Treatment of Three Forms of MPS and CLN2 Disease, a Form of Batten Disease
Feb 4, 2019

SAN RAFAEL, Calif., Feb. 4, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company will present data in four platform and 10 poster presentations at the 15th Annual WORLDSymposiumTM being held February 4-8, 2019 in Orlando, Florida.

BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

"We look forward to the WORLDSymposium every year as it is an unprecedented forum to share and discuss pioneering research for the rare disease community," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "In addition to exploring new potential therapies in areas of great unmet medical need, we also continue to examine our treatments long after they have been approved by regulatory authorities. We look forward to sharing our findings."

Listing of BioMarin Posters and Presentations at the 15th Annual WORLDSymposiumTM

Platform Presentations
Presentations will be held in the Regency Ballroom U.

Title

Presenter

ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

 

Presentation: February 7 at 1:00 – 1:15 PM

Maureen Cleary,

Great Ormond Street Hospital

Insights into Sanfilippo syndrome provided by the ConnectMPS worldwide online registry

 

Presentation: February 7 at 2:15 – 2:30 PM

Edwin Chavez-Cintora,

BioMarin Pharmaceutical

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3-year update from an ongoing multicenter extension study

 

Presentation: February 7 at 3:30 – 3:45 PM

Angela Schulz,

University Medical Center Hamburg-Eppendorf

Evidence-based, expert-agreed recommendations for the management of patients with MPS IVA/VI: Recommendations to replace the specific missing enzyme

 

Presentation: February 7 at 4:15 – 4:30 PM

Christian J. Hendriksz,

Steve Biko Academic Hospital, University of Pretoria

Poster Presentations

MPS IVA & VI


Title

Authors

Impact of two-year elosulfase alfa treatment on patient-reported outcomes in patients with Morquio A: Results from an English Managed Access Agreement

 

Presentation: February 5 at 4:30 – 6:30 PM
Poster/Presentation: #6

Adam J, Cleary M, Hendriksz CJ, Hiwot TG, Hughes D, Jones SA, Jovanovic A, Morrison A, Murphy E, Vijay S

Enzyme replacement therapy in patients with mucopolysaccharidosis type VI:

Updated findings from the MPS VI Clinical Surveillance Program

 

Presentation: February 5 at 4:30 – 6:30 PM
Poster/Presentation: #146

Harmatz P, Lampe C, Parini R, Sharma R, Leão-Teles E, Johnson J, Sivam D, Sisic Z

Analysis of the impact of cell-based neutralizing antibody status on long-term efficacy of elosulfase alfa

 

Presentation: February 5 at 4:30 – 6:30 PM
Poster/Presentation: #LB-25

Hock B, Slasor P, Bray L, Lau K, Melton A, Jesaitis L, Qi Y, Solari P

 

MPS


Title

Authors

Evidence-based, expert-agreed recommendations for the management of patients with MPS IVA/VI

 

Presentation: February 5 at 4:30 – 6:30 PM

Poster/Presentation: #155

Hendriksz C, Giugliani R, Harmatz P, Scarpa M

Transition from pediatric to adult care in patients with mucopolysaccharidosis (MPS)

 

Presentation: February 5 at 4:30 – 6:30 PM

Poster/Presentation: #207

Lampe C, McNelly B, Geavorkian A, Hendriksz C, Lobzhanidze T, Perez-Lopez J, Vashakmadze N, del Toro M

Critical care situations in adult patients with mucopolysaccharidosis (MPS)

 

Presentation: February 6 at 4:30 – 6:30 PM

Poster/Presentation: #340

Stepien K, Geavorkian A, Hendriksz C, Lobzhanidze T, Perez-Lopez J, del Toro M, Vashakmadze N, Lampe C

LC-MS/MS-based glycosaminoglycan measurement: Expert considerations on

methods and use in screening, diagnosis and management of patients with mucopolysaccharidoses

 

Presentation: February 5 at 4:30 – 6:30 PM

Poster/Presentation: #LB-18

Fuller M, Auray-Blais C, Burke D, Burlina A, Chuang C-K, Cólon C, Crawford B, Fletcher J, Gelb M, Heales S, Izzo E, Kubaski F, la Marca G, Lawrence R, Matern D, Merry C, Miller N, Pasquali M, Wood T, Harmatz P

 

MPS IIIB


Title

Authors

ICV-administered BMN 250 (NAGLU-IGF2) is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)

 

Presentation: February 5 at 4:30 – 6:30 PM

Poster/Presentation: #67

Cleary M, Muschol N, Couce ML, Harmatz P, Lee J, Lin S, Okur I, Ezgu F, Peters H, Solano Villarreal M, Shaywitz AJ, Cahan H, Grover A, Melton A, Smith L, Maricich SM, de Castro Lopez MJ

Understanding Sanfilippo signs, symptoms and physician testing patterns: Insights from the Simply Test for MPS™ enzyme-panel program (ST4MPS)

 

Presentation: February 5 at 4:30 – 6:30 PM

Poster/Presentation: #142

Gurnon S, Pan W, Chu D, Ly C, Wood T, Pollard L

Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)

 

Presentation: February 6 at 4:30 – 6:30 PM

Poster/Presentation: #335

Solano Villarreal M, Harmatz P, Cleary M, de Castro Lopez MJ, Lee J, Lin S, Okur I, Ezgu F, Muschol N, Peters H, Shaywitz AJ, Cahan H, Grover A, Maricich SM, Melton A, Smith L, Couce ML

About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com.  Information on such website is not incorporated by reference into this press release.

Links to Full Prescribing Information on BioMarin Products

www.Brineura.com 

www.Naglazyme.com

www.VIMIZIM.com 

 

Contact:




Investors:

Media:

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

 

SOURCE BioMarin Pharmaceutical Inc.


BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Palynziq™
Brineura™
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

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