Researchers need help from people with Ataxia to look for a cure and develop new treatments. Here are things that you can do to be a part of the fight against Ataxia.
Patient Registries are essential tools for Ataxia researchers. The purpose of a patient registry is to advance the development of treatments for a disease by connecting researchers with those who are affected with that disease. If you or a family member are affected by Ataxia or are at risk for Ataxia, we recommend that you sign up on the appropriate patient registry listed below (you can be included on more than one patient registry.)
Ataxia Patient Registry at CoRDS The CoRDS Registry was established to facilitate the establishment of rare disease registries for all rare diseases with a specific questionnaire for those with any type of ataxia or those at risk for Ataxia.
CoRDS Registry at Sanford Research Video
Friedreich’s Ataxia Research Alliance Patient Registry for those with Friedreich’s ataxia.
The Fragile X Research Registry is for families who have individuals with Fragile X-associated tremor/ataxia syndrome (FXTAS) in the United States.
Autosomal recessive spastic ataxia of Charlevoix-Saguenay ARSACS The purpose of this website is to create a new database for the SACSIN gene mostly reported in the province of Quebec, Canada.
Ataxia-Telangiectasia (A-T) Children’s Project Family Data Base is to help A-T families stay in touch with the A-T Children’s Project for information about clinical studies.
Participating in a clinical trial is one way to take an active role in furthering understanding and treatment of Ataxia. It is also a way to get access to new treatment options before they are widely available. However, participating in a clinical trial is a deeply personal choice to make. Look into risks and benefits of each trial before enrolling. The following links are great resources for finding research studies that are looking for volunteer participants.
The following links will provide information about participating in a clinical trial.
During the COVID-19 pandemic, some in-person research studies are not being scheduled at this time. However, some studies are currently recruiting research participants for their studies and scheduling future appointments. Safety of research participants is of the utmost importance and appropriate guidelines are being followed. Please contact the institute listed for more details. Thank you.
The National Ataxia Foundation is committed to providing information to those affected by Ataxia including the availability of research studies and clinical trials for the study of Ataxia.
Below you will find clinical trials and research opportunities for those with various types of Ataxia. These trials have been approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits.
DRPLA Patients and Families Needed for Important Research Study
20 DRPLA patients and families in the UK/Europe are needed to work with Ataxia UK and CureDRPLA to develop DRPLA blood-based biomarkers of disease progression. If you are a DRPLA family who may be interested in being part of this IRB approved study, or a physician who sees patients with DRPLA, please email:
Dr. Henry Houlden
Professor of Neurology and Neurogenetics,
UCL Institute of Neurology and
The National Hospital for Neurology and Neurosurgery,
Queen Square, London WC1N 3BG.
One of the challenges in developing a treatment for a rare disease, such as Ataxia, is the lack of natural history studies. 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.With the recent funding by the National Ataxia Foundation of the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA), a natural history study will begin for SCAs 1, 2, 3, 6 and 7 (and SCA 8 and 10 at some sites). The institutions and principle investigators that are in the CRC-SCA are listed below:
Dr. Sheng Han Kuo
Lead Coordinator:
Nadia Amokrane
Na2855@cumc.columbia.edu
Dr. George “Chip” Wilmot
Lead Coordinator:
Carole Seeley
carole.seeley@emory.edu
Dr. Tetsuo Ashizawa
Lead Coordinator:
Titilayo Olubajo
tolubajo@houstonmethodist.org
Drs. Liana Rosenthal and Chiadikaobi Onyike
Lead Coordinator:
Ann Fishman
ann.fishman@jhu.edu
Dr. Jeremy Schmahmann
Lead Coordinator:
Jason MacMore
jmacmore@mgh.harvard.edu
Dr. Puneet Opal
Lead Coordinator:
Alejandro Hernandez
alejandro.hemandez1@
northwestern.edu
Dr. Susan Perlman
Lead Coordinator:
Aaron Fisher
ADFisher@mednet.ucla.edu
Drs. Cameron Dietiker and Daniel Geschwind
Lead Coordinator:
Daven Crossland
Daven.Crossland@ucsf.edu
Dr. Christopher Gomez
Lead Coordinator:
Hannah Casey
hannahcasey@uchicago.edu
Dr. SH Subramony
Lead Coordinator:
Jamie Bolling Jamie.Bolling@neurology.ufl.edu
Drs. Henry Paulson and Vikram Shakkottai
Lead Coordinator:
Tasha Kaiser
kaisert@med.umich.edu
Dr. Khalaf Bushara
Lead Coordinator:
Diane Hutter
hutte019@umn.edu
Dr. Theresa Zesiewicz
Lead Coordinator:
Paige Ricketts
pricketts@usf.edu
Dr. Stefan Pulst
Lead Coordinator:
Pattie Figueroa
karlaf@genetics.utah.edu
The success of the natural history study is dependent on participation by individuals who have those forms of SCA. If you have SCA 1, 2, 3, 6 or 7 (and SCA 8 and 10 at some sites) and are a patient at one of those sites or can travel to a site, please contact the clinic office for more information. You may also contact Sue Hagen at NAF at 763-231-2742 or susan@ataxia.org for more information.
Donating tissue for medical research is an important and deeply personal decision. Proper planning can help ensure that wishes are accounted for and honored at the time of your or a loved one’s passing. For additional information on tissue donation, contact the National Ataxia Foundation at naf@ataxia.org or call 763-553-0020. Thank you for considering this important legacy gift.
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