BMRN BioMarin Pharmaceutical Inc

Title

Authors

Preliminary safety and pharmacodynamics response data from a phase 1/2 study of ICV BMN 250, a novel enzyme replacement therapy for the treatment of Sanfilippo syndrome type B (MPS IIIB)

Parallel Session 1: Wednesday, September 6 from 12:00-12:15

Steve Maricich, MD, PhD, BioMarin Pharmaceutical Inc. Novato, CA, USA

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter extension study

Parallel Session 1: Wednesday, September 6 from 12:00-12:15

Marina Trivisano, MD, Bambino Gesú Children's Hospital, IRCCS, Rome, Italy

Phase 2 long-term pegvaliase treatment for adults with phenylketonuria: updated year 5 safety and efficacy data from the PAL-003 extension

Parallel Session 7: Wednesday, September 6 from 14:45-15:00

Jerry Vockley, MD, PhD, Children's Hospital of Pittsburgh and University of Pittsburgh, Pittsburgh, PA, USA

MPS

Title

Authors

Design and rationale of ongoing observational and treatment studies for BMN 250, a novel enzyme replacement therapy for Sanfilippo syndrome type B (MPS IIIB)

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 597

Shaywitz A, Maricich S, Yu H, Kent S

Presenting signs and symptoms of MPS: results of an international physician survey

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 649

Clarke L, Ellaway C, Foster H, Giugliani R, Goizet C, Goring S, Hawley S, Jurecki E, Khan Z, Lampe C, Martin K, McMullen S, Mitchell J, Mubarack F, Muenzer J, Sivri S, Stewart F, Tylki-Szymanska A, White K, Wijburg F

Presenting signs and symptoms of MPS: results of systematic literature analysis

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 662

Mubarack F, Clarke L, Ellaway C, Foster H, Giugliani R, Goizet C, Goring S, Hawley S, Jurecki E, Khan Z, Lampe C, Martin K, McMullen S, Mitchell J, Muenzer J, Sivri S, Stewart FJ, Tylki-Szymanska A, White K, Wijburg F

Mucopolysaccharidosis type VI (MPS VI) and molecular analysis: a review of published classified variants in the ARSB gene

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 674

Tomanin R, Karageorgos L, AlSayed M, Bailey M, Izzo E, Miller N, Sakuraba H, Zanetti A, Hopwood JJ

Impact of elosulfase alfa treatment on patient-reported outcomes in Morquio A syndrome: results from the first year of an English managed access agreement

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 807

Lavery C, Jones SA, Hughes D, Murphy E, Jovanovic A, Hendriksz C, Cleary M, Hiwot T, Vijay S

CLN2

Title

Authors

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter extension study

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 787

Trivisano M, Schulz A, Specchio N, Gissen P, de los Reyes E, Cahan H, Slasor P, Ajayi T, Jacoby D

PKU

Title

Authors

Phase 3 PRISM-2 long-term extension study evaluating efficacy and safety of pegvaliase for treatment of adults with phenylketonuria

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 285

Vockley J, Levy H, Amato S, Zori R, Thomas J, Burton B, Harding C, Longo N, Posner J, Bilder D, Olbertz J, Gu Z, Lau K, Lin M, Larimore K, Dimmock D

Phase 2 long-term pegvaliase treatment for adults with phenylketonuria: updated year 5 safety and efficacy data from the PAL-003 extension

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 288

Zori R, Levy H, Longo N, Lau K, Rosen O, Li M, Larimore K, Decker C, Weng H, Vockley J

Seventh interim analysis of the Kuvan^® Adult Maternal Paediatric European Registry  (KAMPER): interim results in BH4 deficiency patients

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 295

Van Spronsen FJ, Burlina A, Alm J, Belanger-Quintana A, Feillet F, Lagler FB, Muntau AC, Trefz FK, Jurecki ER, Kittus R, Alvarez I

Seventh interim analysis of the Kuvan^® Adult Maternal Paediatric European Registry  (KAMPER): interim results in phenylketonuria patients

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation:296

Van Spronsen FJ, Muntau AC, Lagler FB, Feillet F, Alm J, Burlina A, Belanger-Quintana A, Alvarez  I, Lilienstein J, Jurecki E, Trefz FK

Interim analysis of the phenylketonuria (PKU) patients enrolled in the PKUDOS registry

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation:297

Lilienstein J, Burton B, Grant M, Ficicioglu C, Kopesky J, Nguyen-Driver MD, Moore C, Jurecki E, Longo N

An interim analysis of the Kuvan^®Adult Maternal Paediatric European Registry  (KAMPER) and phenylketonuria developmental outcomes and safety (PKUDOS) registries: pregnancies

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 298

Feillet F, Ficicioglu C, Lagler FB, Longo N, Alm J, Muntau AC, Burlina A, Belnager-Quintana A, Trefz FK, Kittus R, Jurecki E, Alvarez I, Lilienstein J, Burton B

Estimation of resource use and quality of life in phenylketonuria (PKU) patients in Ireland

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 262

Pastores G, Bracken J, Hughes J, Rogers Y, Stenson C, Clark A, Lloyd A, Alvarez I, Jain M, Kha A, Monavari A

Short-term biological variance of PHE in patients with phenylketonuria

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 250

Potter M, Pendar A, Langley E, Geraghty M

Amino acid level correlations between tandem mass spectrometry and ultra-performance liquid chromatography and the clinical relevance for phenylketonuria management

Presentation: Wednesday, September 6 from 17:30-20:00

Poster/Presentation: 251

Potter M, Pendar A, Langley E, Geraghty M

Investors:

Media:

Traci McCarty 

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451