BioMarin Announces 14 Presentations at 15th Annual WORLDSymposium(TM) 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 15(th) Annual WORLDSymposium(TM) being held February 4-8, 2019 in Orlando, Florida.
"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 15(th) Annual WORLDSymposium(TM)
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)
Maureen Cleary,
Great Ormond Street Hospital
Presentation:
February 7 at 1:00
- 1:15 PM
---
Insights into
Sanfilippo syndrome
provided by the
ConnectMPS
worldwide online
registry
Edwin Chavez-Cintora,
BioMarin Pharmaceutical
Presentation:
February 7 at 2:15
- 2:30 PM
---
Persistent treatment
effect of
cerliponase alfa in
children with CLN2
disease: A 3-year
update from an
ongoing multicenter
extension study
Angela Schulz,
University Medical Center Hamburg-
Eppendorf
Presentation:
February 7 at 3:30
- 3:45 PM
---
Evidence-based,
expert-agreed
recommendations for
the management of
patients with MPS
IVA/VI:
Recommendations to
replace the
specific missing
enzyme
Christian J. Hendriksz,
Steve Biko Academic Hospital,
University of Pretoria
Presentation:
February 7 at 4:15
- 4:30 PM
---
Poster Presentations
MPS IVA & VI
---
Title Authors
---
Impact of two-year Adam J, Cleary M, Hendriksz
elosulfase alfa treatment on CJ, Hiwot TG, Hughes D,
patient-reported outcomes Jones SA, Jovanovic A,
in patients with Morquio A: Morrison A, Murphy E,
Results from an English Vijay S
Managed Access Agreement
Presentation: February 5 at
4:30 - 6:30 PM
Poster/Presentation: #6
---
Enzyme replacement therapy in Harmatz P, Lampe C, Parini
patients with R, Sharma R, Leão-Teles
mucopolysaccharidosis type E, Johnson J, Sivam D,
VI: Sisic Z
Updated findings from the MPS
VI Clinical Surveillance
Program
Presentation: February 5 at
4:30 - 6:30 PM
Poster/Presentation: #146
---
Analysis of the impact of Hock B, Slasor P, Bray L,
cell-based neutralizing Lau K, Melton A, Jesaitis
antibody status on long- L, Qi Y, Solari P
term efficacy of elosulfase
alfa
Presentation: February 5 at
4:30 - 6:30 PM
Poster/Presentation: #LB-25
---
MPS
---
Title
Authors
---
Evidence-based, expert- Hendriksz C, Giugliani R, Harmatz P,
agreed recommendations Scarpa M
for the management of
patients with MPS IVA/VI
Presentation: February 5
at 4:30 - 6:30 PM
Poster/Presentation: #155
---
Transition from pediatric Lampe C, McNelly B, Geavorkian A,
to adult care in patients Hendriksz C, Lobzhanidze T, Perez-
with Lopez J, Vashakmadze N, del Toro M
mucopolysaccharidosis
(MPS)
Presentation: February 5
at 4:30 - 6:30 PM
Poster/Presentation: #207
---
Critical care situations Stepien K, Geavorkian A, Hendriksz
in adult patients with C, Lobzhanidze T, Perez-Lopez J,
mucopolysaccharidosis del Toro M, Vashakmadze N, Lampe C
(MPS)
Presentation: February 6
at 4:30 - 6:30 PM
Poster/Presentation: #340
---
LC-MS/MS-based Fuller M, Auray-Blais C, Burke D,
glycosaminoglycan Burlina A, Chuang C-K, Cólon C,
measurement: Expert Crawford B, Fletcher J, Gelb M,
considerations on Heales S, Izzo E, Kubaski F, la
Marca G, Lawrence R, Matern D,
Merry C, Miller N, Pasquali M, Wood
T, Harmatz P
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
---
MPS IIIB
---
Title Authors
---
ICV-administered BMN 250 (NAGLU- Cleary M, Muschol N,
IGF2) is well tolerated and Couce ML, Harmatz P, Lee
reduces heparan sulfate J, Lin S, Okur I, Ezgu
accumulation in the CNS of F, Peters H, Solano
subjects with Sanfilippo syndrome Villarreal M, Shaywitz
type B (MPS IIIB) AJ, Cahan H, Grover A,
Melton A, Smith L,
Maricich SM, de Castro
Lopez MJ
Presentation: February 5 at 4:30 -
6:30 PM
Poster/Presentation: #67
---
Understanding Sanfilippo signs, Gurnon S, Pan W, Chu D,
symptoms and physician testing Ly C, Wood T, Pollard L
patterns: Insights from the Simply
Test for MPS(TM) enzyme-panel
program (ST4MPS)
Presentation: February 5 at 4:30 -
6:30 PM
Poster/Presentation: #142
---
Natural history data for young Solano Villarreal M,
subjects with Sanfilippo syndrome Harmatz P, Cleary M, de
type B (MPS IIIB) 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
Presentation: February 6 at 4:30 -
6:30 PM
Poster/Presentation: #335
---
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
Contact:
Investors:
Media:
Traci McCarty Debra Charlesworth
BioMarin Pharmaceutical
Inc. BioMarin Pharmaceutical Inc.
(415) 455-7558
(415) 455-7451
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