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Office
707 SW Gaines St
Portland, OR 97239Phone+1 503-494-7859Fax+1 503-494-4447
Education & Training
- University of Wisconsin Hospitals and ClinicsResidency, Pediatrics, 1987 - 1990
- University of Washington School of MedicineClass of 1987
Certifications & Licensure
- OR State Medical License 1999 - 2025
- ID State Medical License 2004 - 2024
- WI State Medical License 1988 - 1999
- American Board of Medical Genetics and Genomics Clinical Biochemical Genetics
- American Board of Medical Genetics and Genomics Clinical Genetics and Genomics
Clinical Trials
- Longitudinal Study of Urea Cycle Disorders Start of enrollment: 2006 Feb 01
- Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders Start of enrollment: 2010 Dec 01
- Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder Start of enrollment: 2011 Sep 01
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Publications & Presentations
PubMed
- 3 citationsPegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program.Cary O Harding, Nicola Longo, Hope Northrup, Stephanie Sacharow, Rani Singh
Molecular Genetics and Metabolism Reports. 2024-06-01 - 14 citationsSafety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2 months to 2 yearsSusan A. Berry, Nicola Longo, George A. Diaz, Shawn E. McCandless, Wendy E. Smith
Molecular Genetics and Metabolism. 2017-11-01 - 126 citationsIntracerebral accumulation of glutaric and 3-hydroxyglutaric acids secondary to limited flux across the blood-brain barrier constitute a biochemical risk factor for ne...Sven W. Sauer, Jürgen G. Okun, Gert Fricker, Anne Mahringer, Ines Müller
Journal of Neurochemistry. 2006-05-01
Press Mentions
- BioMarin Pharmaceutical : , Pioneer in Rare Disease Treatments for Phenylketonuria, Receives FDA Approval of Label ExpansionOctober 15th, 2020
- FDA Approves New Drug Tested at OHSU for Phenylketonuria, or PKU - Portland Business JournalMay 25th, 2018
- BioMarin Sinks Despite Meeting Primary EndpointMarch 21st, 2016
Grant Support
- Augmented Phenylalanine Clearance By Muscles As Novel Therapy For PhenylketonuriaEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2012
- AAV2/8 Vector-Mediated Liver Gene Therapy For Phenylketonuria (PKU)National Institute Of Diabetes And Digestive And Kidney Diseases2007–2009
- Liver Gene Therapy For Murine Phenyketonuria (PKU)National Institute Of Diabetes And Digestive And Kidney Diseases2006
- Intravenous Non-Viral Gene Therapy For Phenylketonuria (PKU)National Institute Of Diabetes And Digestive And Kidney Diseases2006
- Stem Cell-Mediated Liver Repopulation For Murine PKUNational Heart, Lung, And Blood Institute2005–2006
- DHA Triglyceride Supplementation In Lchadd PatientsNational Center For Research Resources2004–2005
- Metabolic Engineering As Therapy For Murine PKUNational Institute Of Diabetes And Digestive And Kidney Diseases2001–2002
- Docosahexaenoic Acid Triglyceride Supplementation In LchaddNational Center For Research Resources2000–2002
- DHA Triglyceride Supplementation In Long Chain Dehydrogenase Deficiency (LCHADD)National Center For Research Resources1998–2002
- Heterologous Gene Therapy For MetabolismNational Institute Of Diabetes And Digestive And Kidney Diseases1999–2000
- Aav-Mediated Gene Therapy--Inborn Errors Of MetabolismNational Institute Of Diabetes And Digestive And Kidney Diseases1999
- Heterologous Gene Therapy For MetabolismNational Institute Of Diabetes And Digestive And Kidney Diseases1996–1999
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