Sep 5, 2018 SOURCE: PR NewsWire
BioMarin Announces 20 Poster Presentations at Society for the Study of Inborn Errors of Metabolism (SSIEM) Symposium 2018
SAN RAFAEL, Calif., Sept. 5, 2018 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the company will present 20 poster presentations at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Symposium 2018, which will be held on September 4-7 in Athens, Greece.
Listing of Posters and Presentations at Society for the Study of Inborn Errors of Metabolism 2018 Symposium
Poster Presentations
CLN2 Disease
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Title Authors
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Monitoring neuropsychological function Nickel M, Augustine E, Adams H,
in CLN2 disease Baron I, Bjoraker K, Cohen-
Pfeffer J, Delaney K, Elmerskog B,
Newsom-Davis I, Rust S, Shapiro
E, Tøssebro A, Schulz A
Poster: #345
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Experiences of CLN2 disease diagnosis Burke D, Church H, Jackson M,
in the UK Powers V, Tylee K
Poster: #357
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Intracerebroventricular cerliponase Schulz A, de los Reyes E, Specchio
alfa in children with CLN2 disease: N, Gissen P, Cahan H, Slasor P,
interim results from an ongoing Ajayi T, Jacoby D
multicenter, multinational extension
study
Poster: #367
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Phenylketonuria (PKU)
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Title Authors
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Burden of illness in adult patients Rutsch F, Muntau AC, Alvarez I,
with phenylketonuria and associated Lane P, Altevers J, Kohlscheen KM,
comorbidities - a retrospective Jacob C, Jain M, Schroeder C,
database study in Germany
Jha A, Treftz F
Poster: #095
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Efficacy & safety of sapropterin in PKU Rutsch F, Burlina A, Eyskens F,
patients <4 yrs: 3-year extension of Freisinger P,
SPARK open-label, randomized phase 3b
trial
De Laet C, Leuzzi V, Sivri HS,
Vijay S, Bal MO, Gramer G,
Pazdírková R, Cleary M, Lotz-
Havla AS, Mould DR, Lane P,
Alvarez I, Muntau A
Poster: #097
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Prevalence of comorbidities among Burton B, Cederbaum S, Jurecki E,
phenylketonuria patients - a Lilienstein J, Alvarez I, Cohen-
retrospective study of US health Pfeffer J, Irwin D, Levy H, Rohr
insurance claims data
Poster: #098
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The burden of illness in adults with Burton B, Longo N, Stuy M, Vockley
phenylketonuria (PKU): interim J, van Backle J, Lane P, Alvarez
analysis of a cross-sectional study I, Lilienstein J, Jurecki E
Poster: #099
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International best practice Bhattacharya K, Adams D, Belanger-
recommendations for the evaluation of Quintana A, Bushueva T, Cerone R,
responsiveness to sapropterin Chien N, Chiesa A, Coskun T, De la
dihydrochloride in patients with Heras Montero J, Feillet F, Katz
phenylketonuria R, Lagler F, Muntau A, Piazzon F,
Rohr F, Spronsen F, Vargas P,
Wilcox G
Poster: #117
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Phase 3 PRISM clinical trials Thomas J, Levy H, Amato S, Vockley
evaluating efficacy and safety of G, Zori R, Dimmock C, Harding C,
pegvaliase for treatment of adults Bilder D, Weng HH, Olbertz J,
with phenylketonuria Merilainen M, Rosen O, Gupta S, Gu
K, Larimore K, Northrup H
Poster: #122
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Long-term safety of induction, Burton B, Harding C, Thomas J,
titration, and maintenance dosing of Longo N, Posner J, Dimmock D, Zori
pegvaliase treatment in adults with R, Weng H, Olbertz J, Gershman E,
phenylketonuria Rosen O, Gupta S, Jones S, Gu K,
Vockley J
Poster: #123
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Characterization of Harding C, Thomas J, Burton B, Zori
hypophenylalaninemia in pegvaliase R, Dimmock D, Vockley J, Weng HH,
treated adults with PKU Olbertz J, Gershman A,
Rosen O, Jones S, Li M, Longo N
Poster: #124
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An interim analysis of the KAMPER and Feillet F, Ficicioglu C, Lagler FB,
PKUDOS registries: efficacy and safety Longo N, Alm J, Muntau AC, Burlina
of sapropterin before and during A, Belanger-Quintana A, Trefz FK,
pregnancy Kittus R, Jurecki E, Alvarez I,
Lilienstein J, Burton B
Poster: #125
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A meta-analysis of growth outcomes in Muntau A, Feillet F, Burton B,
phenylketonuria patients treated with McDonald A, Wessel A, Alvarez I,
phenylalanine-restricted diet + Lilienstein J, Lane P, Jurecki E,
sapropterin Longo N
Poster: #126
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Mucopolysaccharidosis (MPS)
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Title Authors
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Natural history data for young subjects Harmatz P, Cleary M, de Castro
with Sanfilippo syndrome type B (MPS Lopez MJ, Lee J, Lin S, Okur I,
IIIB) Ezgu F, Muschol N, Peters H,
Solano Villarreal MS, Shaywitz AJ,
Cahan H, Grover A, Maricich SM,
Melton A, Smith L, Couce ML
Poster: #315
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ICV-administered BMN 250 (NAGLU-IGF2) Lin S, Cleary M, Couce ML, de
is well tolerated and reduces heparan Castro Lopez MJ, Harmatz P, Lee J,
sulfate accumulation in the CNS of Okur I, Ezgu F, Peters H,
subjects with Sanfilippo syndrome type Villarreal MS, Shaywitz AJ, Cahan
B (MPS IIIB) H, Grover A, Maricich SM, Melton
A, Smith L, Muschol N
Poster: #316
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Critical care situations in patients Stepien K, Geavorkian A, Hendriksz
with mucopolysaccharidosis (MPS) C, Lobzhanidze T, Perez-Lopez J,
del Toro M, Vashakmadze N, Lampe C
Poster: #321
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Transition from paediatric to adult Lampe C, McNelly B, Geavorkian A,
care in patients with Hendriksz C, Lobzhanidze T, Perez-
mucopolysaccharidosis (MPS) Lopez J, Vashakmadze N,
del Toro M
Poster: #324
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Mucopolysaccharidosis VI enzyme Harmatz P, Lampe C, Teles EL,
replacement therapy outcomes across Parini R, Sharma R, Sivam D,
the disease spectrum: findings from Johnson J, Hawley S, Sisic Z
the MPS VI clinical surveillance
program
Poster: #362
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Enzyme replacement therapy in patients Harmatz P, Lampe C, Leão-Teles E,
with mucopolysaccharidosis VI: updated Parini R, Sharma R, Sivam D,
findings from the MPS VI clinical Johnson J, Sisic Z
surveillance program
Poster: #363
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Mucopolysaccharidosis VI enzyme Lampe C, Harmatz, Leão-Teles E,
replacement therapy initiated in Parini R, Sharma R, Sivam D,
adulthood: findings from the MPS VI Johnson J, Hawley S, Sisic Z
clinical surveillance program
Poster: #364
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About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare disorders. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates.
For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
Contact:
Investors: Media:
Traci McCarty Debra Charlesworth
BioMarin Pharmaceutical Inc. BioMarin Pharmaceutical Inc.
(415) 455-7558 (415) 455-7451
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