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Office
60 Leveroni Court
Novato, CA 94949Phone+1 415-483-8800- Is this information wrong?
Education & Training
- David Geffen School of Medicine at UCLAClass of 1989
Certifications & Licensure
- CA State Medical License 1990 - 2020
Publications & Presentations
PubMed
- 10 citationsThe multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseasesP. K. Tandon, Emil D. Kakkis> ;Orphanet Journal of Rare Diseases. 2021 Apr 19
- 5 citationsDisease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders.Hanns Lochmüller, Antonio Nino Ramirez, Emil D. Kakkis> ;Orphanet Journal of Rare Diseases. 2021 Mar 20
- 7 citationsGalactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of conceptJaclyn Cadaoas, Huimin Hu, Gabrielle Boyle, Elida Gomero, Rosario Mosca, Kartika Jayashankar, Mike Machado, Sean Cullen, Belle Guzman, Diantha van de Vlekkert, Ida Ann...> ;Molecular Therapy. Methods & Clinical Development. 2021 Mar 12
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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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