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UCSD ACTRI, 9452 Medical Center Drive, Ste 2W-108La Jolla, CA 92037
Phone+1 206-909-5475
Fax+1 858-657-8557
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Education & Training
Clinical Center at the National Institutes of HealthResidency, Neurology, 1984 - 1986
Mass General Brigham/Brigham and Women's Hospital/Massachusetts General Hospital/Harvard Medical SchoolResidency, Neurology, 1983 - 1984
University of California (San Diego) Medical CenterInternship, Transitional Year, 1979 - 1980
Mayo Clinic College of MedicineClass of 1979
Certifications & Licensure
CA State Medical License 2011 - 2027
WA State Medical License 1985 - 2013
American Board of Psychiatry and Neurology Neurology- American Board of Psychiatry and Neurology Clinical Neurophysiology
- American Board of Psychiatry and Neurology Neuromuscular Medicine
Awards, Honors, & Recognition
- Super Doctor SuperDoctors.com
- Top MD Consumers Checkbook
Clinical Trials
- Mexiletine for the Treatment of Muscle Cramps in ALS Start of enrollment: 2013 May 01
- A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS) Start of enrollment: 2021 Sep 02
Roles: Principal Investigator
Publications & Presentations
PubMed
- Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics.Sarah Mizielinska, Guillaume M Hautbergue, Tania F Gendron, Marka van Blitterswijk, Orla Hardiman
The Lancet. Neurology. 2025-03-01 - 1 citationsCK1δ/ε-mediated TDP-43 phosphorylation contributes to early motor neuron disease toxicity in amyotrophic lateral sclerosis.Vivian I Ko, Kailee Ong, Deborah Y Kwon, Xueying Li, Alicia Pietrasiewicz
Acta Neuropathologica Communications. 2024-12-04 - Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised,...Leonard H van den Berg, Jeffrey D Rothstein, Pamela J Shaw, Suma Babu, Michael Benatar
The Lancet. Neurology. 2024-09-01
Journal Articles
- Transcriptome–Pathology Correlation Identifies Interplay Between TDP-43 and the Expression of Its Kinase CK1E in Sporadic ALSJohn Ravits, MD, Acta Neuropathologica
Press Mentions
ALS-Causative Mutations in FUS/TLS Confer Gain- and Loss-of-Function by Altered Association with SMN and U1-snRNPJanuary 27th, 2020
Researchers Describe New ALS Biomarkers, Potential New Drug TargetsJuly 16th, 2019
Restoring Levels of Membralin Protein May Extend Survival in ALS, Mouse Study SuggestsJune 12th, 2019- Join now to see all
Grant Support
- Microgenomic Studies Of ALSNational Institute Of Neurological Disorders And Stroke2006–2007
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