BIOMARIN PHARMACEUTICAL INC. 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 | |
Title | Authors |
Monitoring neuropsychological function in CLN2 disease
Poster: #345 | Nickel M, Augustine E, Adams H, Baron I, Bjoraker K, Cohen-Pfeffer J, Delaney K, Elmerskog B, Newsom-Davis I, Rust S, Shapiro E, Tøssebro A, Schulz A |
Experiences of CLN2 disease diagnosis in the UK
Poster: #357 | Burke D, Church H, Jackson M, Powers V, Tylee K |
Intracerebroventricular cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study
Poster: #367 | Schulz A, de los Reyes E, Specchio N, Gissen P, Cahan H, Slasor P, Ajayi T, Jacoby D |
Phenylketonuria (PKU) | |
Title | Authors |
Burden of illness in adult patients with phenylketonuria and associated comorbidities – a retrospective database study in Germany
Poster: #095 | Rutsch F, Muntau AC, Alvarez I, Lane P, Altevers J, Kohlscheen KM, Jacob C, Jain M, Schroeder C, Jha A, Treftz F |
Efficacy & safety of sapropterin in PKU patients <4 yrs: 3-year extension of SPARK open-label, randomized phase 3b trial
Poster: #097 | Rutsch F, Burlina A, Eyskens F, Freisinger P, 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 |
Prevalence of comorbidities among phenylketonuria patients – a retrospective study of US health insurance claims data
Poster: #098 | Burton B, Cederbaum S, Jurecki E, Lilienstein J, Alvarez I, Cohen-Pfeffer J, Irwin D, Levy H, Rohr |
The burden of illness in adults with phenylketonuria (PKU): interim analysis of a cross-sectional study
Poster: #099 | Burton B, Longo N, Stuy M, Vockley J, van Backle J, Lane P, Alvarez I, Lilienstein J, Jurecki E |
International best practice recommendations for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria
Poster: #117 | Bhattacharya K, Adams D, Belanger-Quintana A, Bushueva T, Cerone R, Chien N, Chiesa A, Coskun T, De la Heras Montero J, Feillet F, Katz R, Lagler F, Muntau A, Piazzon F, Rohr F, Spronsen F, Vargas P, Wilcox G |
Phase 3 PRISM clinical trials evaluating efficacy and safety of pegvaliase for treatment of adults with phenylketonuria
Poster: #122 | Thomas J, Levy H, Amato S, Vockley G, Zori R, Dimmock C, Harding C, Bilder D, Weng HH, Olbertz J, Merilainen M, Rosen O, Gupta S, Gu K, Larimore K, Northrup H |
Long-term safety of induction, titration, and maintenance dosing of pegvaliase treatment in adults with phenylketonuria
Poster: #123 | Burton B, Harding C, Thomas J, Longo N, Posner J, Dimmock D, Zori R, Weng H, Olbertz J, Gershman E, Rosen O, Gupta S, Jones S, Gu K, Vockley J |
Characterization of hypophenylalaninemia in pegvaliase treated adults with PKU
Poster: #124 | Harding C, Thomas J, Burton B, Zori R, Dimmock D, Vockley J, Weng HH, Olbertz J, Gershman A, Rosen O, Jones S, Li M, Longo N |
An interim analysis of the KAMPER and PKUDOS registries: efficacy and safety of sapropterin before and during pregnancy
Poster: #125 | Feillet F, Ficicioglu C, Lagler FB, Longo N, Alm J, Muntau AC, Burlina A, Belanger-Quintana A, Trefz FK, Kittus R, Jurecki E, Alvarez I, Lilienstein J, Burton B |
A meta-analysis of growth outcomes in phenylketonuria patients treated with phenylalanine-restricted diet + sapropterin
Poster: #126 | Muntau A, Feillet F, Burton B, McDonald A, Wessel A, Alvarez I, Lilienstein J, Lane P, Jurecki E, Longo N |
Mucopolysaccharidosis (MPS) | |
Title | Authors |
Natural history data for young subjects with Sanfilippo syndrome type B (MPS IIIB)
Poster: #315 | Harmatz P, Cleary M, de Castro Lopez MJ, Lee J, Lin S, Okur I, Ezgu F, Muschol N, Peters H, Solano Villarreal MS, Shaywitz AJ, Cahan H, Grover A, Maricich SM, Melton A, Smith L, Couce ML |
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)
Poster: #316 | Lin S, Cleary M, Couce ML, de Castro Lopez MJ, Harmatz P, Lee J, Okur I, Ezgu F, Peters H, Villarreal MS, Shaywitz AJ, Cahan H, Grover A, Maricich SM, Melton A, Smith L, Muschol N |
Critical care situations in patients with mucopolysaccharidosis (MPS)
Poster: #321 | Stepien K, Geavorkian A, Hendriksz C, Lobzhanidze T, Perez-Lopez J, del Toro M, Vashakmadze N, Lampe C |
Transition from paediatric to adult care in patients with mucopolysaccharidosis (MPS)
Poster: #324 | Lampe C, McNelly B, Geavorkian A, Hendriksz C, Lobzhanidze T, Perez-Lopez J, Vashakmadze N, del Toro M |
Mucopolysaccharidosis VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program
Poster: #362 | Harmatz P, Lampe C, Teles EL, Parini R, Sharma R, Sivam D, Johnson J, Hawley S, Sisic Z |
Enzyme replacement therapy in patients with mucopolysaccharidosis VI: updated findings from the MPS VI clinical surveillance program
Poster: #363 | Harmatz P, Lampe C, Leão-Teles E, Parini R, Sharma R, Sivam D, Johnson J, Sisic Z |
Mucopolysaccharidosis VI enzyme replacement therapy initiated in adulthood: findings from the MPS VI clinical surveillance program
Poster: #364 | Lampe C, Harmatz, Leão-Teles E, Parini R, Sharma R, Sivam D, Johnson J, Hawley S, Sisic Z |
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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