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Office
10 Center Dr
BETHESDA, MD
Bethesda, MD 20892Phone+1 202-884-2187- Is this information wrong?
Education & Training
National Capital ConsortiumResidency, Pediatrics, 1986 - 1987
University of Texas Health Science Center at HoustonResidency, Pediatrics, 1984 - 1986
McGovern Medical School at UTHealthClass of 1983
Certifications & Licensure
MD State Medical License 1986 - 2025
DC State Medical License 1991 - 2022- American Board of Medical Genetics and Genomics Clinical Genetics
- American Board of Pediatrics Pediatrics
Clinical Trials
- Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders Start of enrollment: 2002 Feb 06
- Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses Start of enrollment: 2004 Jul 01
Publications & Presentations
PubMed
- -Acetyl-l-Leucine and Neurodegenerative Disease.Tifft, C.> ;The New England Journal of Medicine. 2024 Feb 1
- GM1 Gangliosidosis Type II: Results of a 10-Year Prospective Study.D'Souza, P., Farmer, C., Johnston, J., Han, S., Adams, D., Hartman, A., Zein, W., Huryn, L., Solomon, B., King, K., Jordan, C., Myles, J., Nicoli, E., Rothermel, C., A...> ;Medrxiv. 2024 Jan 4
- 1 citationsSialidase NEU3 action on GM1 ganglioside is neuroprotective in GM1 gangliosidosis.Maria L Allende, Y Terry Lee, Colleen Byrnes, Cuiling Li, Galina Tuymetova, Jenna Y Bakir, Elena-Raluca Nicoli, Virginia K James, Jennifer S Brodbelt, Cynthia J Tifft,...> ;Journal of Lipid Research. 2023 Dec 1
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Journal Articles
- Effect of Genetic Diagnosis on Patients with Previously Undiagnosed DiseaseCarlos A Bacino, Vandana Shashi, Euan A Ashley, Cynthia J Tifft, William A Gahl, Brendan H Lee, John J Mulvihill, Rizwan Hamid, Matthew T Wheeler, Jonathan A Bernstein..., The New England Journal of Medicine
Press Mentions
Sio Gene Therapies Announces CSF Reductions in GM1 Ganglioside from Clinical Trial of AXO-AAV-gm1 Gene TherapyMay 13th, 2021- An Experimental Gene Therapy Was Little Alissa's Only Hope. Now, Instead of Certain Death, She Faces an Uncertain FutureApril 25th, 2021
- Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-gm1 Clinical Trial in Patients with GM1 GangliosidosisDecember 2nd, 2020
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Grant Support
- An Exploratory, Open-Label Study Of The Safety AndNational Center For Research Resources2010
- Pharmacokinetics, Safety, And Tolerability Of Zavesca (MIGLUSTAT) In PatientsNational Center For Research Resources2006–2008
- Pathogenesis And Natural History Of The Glycosphingolipd (GSL) StorageNational Center For Research Resources2006
Hospital Affiliations
Walter Reed National Military Medical CenterBethesda, Maryland
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