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National Ataxia Foundation

Research Pipeline

Natural history study and biomarker identification

To accelerate treatment development

Our Mission

To better understand the factors that determine disease progression among Cerebellar Ataxia with the goal of improving the understanding of the disease process, current treatments, and development of disease-modifying therapies

Patients and Families

Learn more about how you can get involved in the CRC-SCA Natural History Study.

Academic Research Community

General information for researchers working with Ataxia.

Interested Industry

Introductory information for industry partners that are exploring Ataxia therapies.

CRC-SCA Site Log In

For PI’s and Coordinators of CRC-SCA Sites.

CRC-SCA Natural History Study

A natural history study collects data that shows how a specific disease progresses in individuals over time. Natural history studies are essential for developing clinical trial designs that will facilitate drug development. You may also hear the terms ‘observational study’ or ‘observational clinical trial’ used to describe a natural history study.

The natural history of the SCAs and factors that may modify them are not completely understood. There is a need to define natural history in geographically distinct areas and develop additional methods to document progression that may be more sensitive and reliable. There is also limited knowledge of factors that may modify symptoms of SCA. 

The CRC-SCA natural history study and biomarker development is growing and adapting during this exciting new era in ataxia research as pharmaceuticals are advancing to therapy development for the ataxias. CRC-SCA plans to add specific clinical end-point assessments and biospecimen collection. This will increase the strength of this study allowing the consortium to partner with stakeholders in a meaningful way that will include patients as research collaborators.

The Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) continues to recruit research participants who have a confirmed diagnosis of SCA 1, 2, 3, 6, 7, 8 or 10. This is an opportunity for anyone in the United States with those forms of SCA at any stage of the disease to participate. Contact the research coordinator at a site near you to learn more about how you might be able to help in Ataxia research efforts to discover a treatment.

Study Leadership

Liana Rosenthal, MD, PhD

Study Co-Chair

Liana Rosenthal, MD, PhD
Johns Hopkins University,
Baltimore, MD

Study Co-Chair

Vikram Shakkottai, MD, PhD
University of Texas,   
Southwestern Medical Center       
Dallas, TX

Sheng Han Kuo, MD

Study Co-Chair

Sheng-Han Kuo, MD
Columbia University,
New York City, NY

A. Fisher

Lead Coordinator

Database Liaison

Ann Fishman

Operations Liaison

Nadia Amokrane

US Site Map

US Sites & Coordinators

Columbia University

Dr. Sheng Han Kuo

Lead Coordinator:
Nadia Amokrane

Emory University

Dr. George “Chip” Wilmot

Lead Coordinator:
Jonna Seppa

Houston Methodist Research Institute

Dr. Tetsuo Ashizawa

Lead Coordinator:
Erika Espinoza

Johns Hopkins University

Drs. Liana Rosenthal and Chiadikaobi Onyike

Lead Coordinator:
Ann Fishman

Massachusetts General

Dr. Jeremy Schmahmann

Lead Coordinator:
Jason MacMore

Northwestern University

Dr. Puneet Opal

Lead Coordinator:
Natasha Cabigon 

University of California - Los Angeles

Dr. Susan Perlman

Lead Coordinator:
Aaron Fisher

University of California - San Francisco

Drs. Cameron Dietiker and Michael Geschwind

Lead Coordinator:
Chelsea Chen

University of Chicago

Dr. Christopher Gomez

Lead Coordinator:
Hannah Casey

University of Florida

Drs. SH Subramony and Matthew Burns

Lead Coordinator:
Grace Lien 

University of Michigan

Dr. Henry Paulson

Lead Coordinator:
Frank Ferrari

University of South Florida

Dr. Theresa Zesiewicz

Lead Coordinator:
Allison Howard

University of Texas, Southwestern Medical Center

Dr. Vikram Shakkottai

Lead Coordinator:
Jan Cameron Watts

Canada Site & Coordinators

University of Montreal

Montréal, Québec, Canada

Dr. Antoine Duquette

Lead Coordinator:
Martine Comeau

Pending Sites: Anticipated Mid-Year 2023

University of Pennsylvania

Dr. Ali Hanedani

Lead Coordinator:
Anne Beckett-Fedarko 

University of Washington

Dr. Marie Davis

Lead Coordinator:
Sarah Simon

Thank you

NAF extends its appreciation to the Gordon and Marilyn Macklin Foundation for awarding NAF a matching grant that provides support for the sites across the country who are recruiting participants in the Natural History Study. This clinical research collects the essential data needed to provide our industry partners information about disease progression which guides them as they work to develop treatments and design clinical trials. This effort is paramount to make the necessary advancements in pursuit of a treatment for Ataxia and, ultimately, a cure. 

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