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Office
2100 Erwin Rd
Durham, NC 27705Phone+1 919-620-4467Fax+1 919-684-0983
Education & Training
- University of WashingtonFellowship, Medical Genetics and Genomics, 1993 - 1996
- University of California (San Francisco)Residency, Pediatrics, 1990 - 1992
- Mayo Graduate SchoolPh.D., Biochemistry, 1990
- Mayo Clinic College of MedicineClass of 1990
- Carleton CollegeB.A., Chemistry, Magna Cum Laude, 1984
Certifications & Licensure
- CA State Medical License 1992 - Present
- NC State Medical License 1999 - 2025
- American Board of Medical Genetics and Genomics Clinical Biochemical Genetics
- American Board of Pediatrics Pediatrics
- American Board of Medical Genetics and Genomics Clinical Genetics and Genomics
Awards, Honors, & Recognition
- America's Best Doctors 2009
- Judith Graham Pool Fellowship National Hemophilia Society, University of Washington, 1993
- March of Dimes Predoctoral Fellowship 1986
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Clinical Trials
- Albuterol in Individuals With Late Onset Pompe Disease (LOPD) Start of enrollment: 2012 Jun 01
- Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease Start of enrollment: 2013 Jun 01
- Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy Start of enrollment: 2013 Sep 01
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Publications & Presentations
PubMed
- 31 citationsA Liver-Specific Thyromimetic, VK2809, Decreases Hepatosteatosis in Glycogen Storage Disease Type Ia.Jin Zhou, Lauren R Waskowicz, Andrea Lim, Xiao Hui Liao, Brian Lian
Thyroid. 2019-08-19 - 26 citationsTreatment outcome of creatine transporter deficiency: international retrospective cohort studyTheodora U. J. Bruun, Sarah Sidky, Anabela O. Bandeira, Francoise-Guillaume Debray, Can Ficicioglu
Metabolic Brain Disease. 2018-02-12 - 23 citationsAntibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe diseaseBaodong Sun, Songtao Li, Andrew Bird, Haiqing Yi, Alex R. Kemper
The Journal of Gene Medicine. 2010-11-01
Journal Articles
- Beta2 agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine pompe diseaseKoeberl DD, Li S, Dai J, Thurberg BL, Bali D, and Kishnani PS, Mol Genet Metab, 1/1/2012
- Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine Pompe diseaseZhang P, Sun B, Osada T, Rodriguiz R, Yang XY, Luo X, Kemper AR, Clay TM, Koeberl DD, Hum Gene Ther, 1/1/2012
- Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus- mediated gene therapySun,B, Young SP, Li P, Di C, Brown T, Salva MZ, Li S, Bird A, Yan Z, Auten R, Hauschka SD, and Koeberl DD, Mol Ther, 1/1/2008
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Press Mentions
- A New Twist on an Old Hormone Leads to First Drug for a Type of Liver DiseaseAugust 12th, 2024
- AskBio Gets $235 Million in Gene Therapy SupportApril 12th, 2019
- AskBio Partners with TPG and Vida VenturesApril 11th, 2019
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Grant Support
- Phase 1/2 Study of clenbuterol for the treatment of Pompe diseaseFood and Drug Administration2013–Present
- Clinical Trial Planning in Pompe DiseaseNational Institutes of Health2013–2014
- Enhanced muscle-targeted gene therapy for Pompe diseaseMuscular Dystrophy Association2012–2014
- Mechanisms For Immune Tolerance In Pompe DiseaseEunice Kennedy Shriver National Institute Of Child Health &Human Development2009–2010
- Gene Delivery To Striated Muscle By Systemic AAV VectorsNational Heart, Lung, And Blood Institute2006–2010
Professional Memberships
- Fellow
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