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Office
60 Leveroni Court
Novato, CA 94949Phone+1 415-483-8800
Education & Training
- David Geffen School of Medicine at UCLAClass of 1989
Certifications & Licensure
- CA State Medical License 1990 - 2020
Publications & Presentations
PubMed
- 141 citationsEfficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controll...Friedrich K. Trefz, Barbara K. Burton, Nicola Longo, Mercedes Martinez Pardo Casanova, Daniel Gruskin
The Journal of Pediatrics. 2009-05-01 - 415 citationsEnzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (lar...James E. Wraith, Lorne A. Clarke, Michael Beck, Edwin H. Kolodny, Gregory M. Pastores
The Journal of Pediatrics. 2004-05-01 - 140 citationsLong-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis IEmil D. Kakkis, Michael F. McEntee, Artur Schmidtchen, Elizabeth F. Neufeld, Daniel A. Ward
Biochemical and Molecular Medicine. 1996-08-01
Press Mentions
- Three Misconceptions About the Accelerated Drug Approval PathwayMay 10th, 2023
- COVID Vaccines Made mRNA a Household Name. How Can It Help in Rare Diseases?December 26th, 2022
- With Data for 11 Patients in, Ultragenyx Ready to Sign Away $75M for Angelman Partner GeneTxJuly 18th, 2022
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Grant Support
- Production Of Beta-Glucuronidase For MPS VIINational Institute Of Diabetes And Digestive And Kidney Diseases2001–2002
- Enzyme Replacement Therapy In Treatment Of Mucopolysaccharidosis INational Center For Research Resources1999–2001
- Enzyme Replacement Therapy In Treatment Of MPS INational Center For Research Resources1999–2001
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