Author: Sue Hagen, Research Services Director
In the 14 years that I have been on staff at the National Ataxia Foundation, I have heard many times that clinical trials for therapy development for Ataxia will start within the next 5 years. And I have seen the Ataxia community patiently and at times, impatiently, waiting for those trials to begin. The good news is that within the last five years, there have been two significant trials sponsored by industry: one for Friedreich’s Ataxia and one for some types of SCA.
However, I think I can speak for those living with Ataxia that more are needed. And the sooner the better. So, I want to share with you, the Ataxia community, how urgently your help is needed to pursue NAF’s vision of: A World Without Ataxia.
Why Your Participation is Essential
In my role at NAF, I participate in meetings with pharmaceutical companies, Ataxia researchers, the NAF Drug Development Collaborative and other groups who are working diligently in research for the development of treatments for Ataxia. They have the brain power, the scientific knowledge, the financial resources to move forward, but more than anything, they need the Ataxia patient population to participate. Drug development cannot take place without patient involvement.
The participation of the patient population cannot be overstated. Below are ways that you can be a part of the process of bringing an approved treatment to the Ataxia community.
- Become a member of NAF – it is free and easy to sign-up at www.ataxia.org/JoinNAF. We currently have 6,590 members. Let’s bring that number up to 10,000! There is strength in numbers.
- Join the CoRDS Ataxia Patient Registry at https://research.sanfordhealth.org/rare-disease-registry.
- Encourage your family members to join NAF and CoRDS.
- Join a support group in your area or attend a virtual support group. Information on support groups is located at www.ataxia.org/support-groups.
- Learn about the Clinical Research Consortium for the Study of Cerebellar Ataxia and if you qualify, contact a Research Coordinator, and ask if you may enroll in the natural history study.
- If you are able and qualify, consider donating biosamples for research. Sometimes this will be a blood draw, but also needed is cerebrospinal fluid (CSF), which is done by a lumbar puncture. This may cause some temporary discomfort, but the value of CSF from patients with certain SCAs is necessary to bring us closer to drug development. (Speak with your physician about whether it is safe for you to undergo a lumbar puncture.)
- If you have participated in a clinical trial or research study, share your story with NAF. Stories can be sent to naf@ataxia.org. Your story may encourage others to participate in research.
Thank you for becoming involved and engaged so that the mission of NAF can move forward: To accelerate the development of treatments and a cure while working to improve the live of those living with Ataxia. Feel free to send me an email if you have any questions: susan@ataxia.org.
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