Trials

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I hope you all are doing well. I was just checking up on ataxia trial news. If you ever want to check out what is going on yourself, google clinicaltrials.gov and just type in SCA6 or whatever disease you are interested in into the search box.

Doing this reminded me that the PRIME-Ataxia exercise study is recruiting. This study allows remote participation (flyer attached).

I also listened to a video about how the first FDA approval of a medicine to help with Freidrich's ataxia came about. In the video, the company scientist described the importance of having a robust disease life history database and patient advocacy.

The FDA recently the FDA announced approval for SKYCLARYS (omaveloxolone) for the treatment of Friedreich’s Ataxia. This is the first and only FDA-approved prescription medicine for Friedreich’s Ataxia (FA). NAF is thrilled about this announcement and proud to have been involved in the process, from clinical trial recruitment to educating our community about the drug development process and how treatments for Ataxia will be accessed. Thank you to all the FA patients who participated in research to make this possible! Read Reata Pharmaceutical’s full press release for more details.

It is disappointing news about the closure of the MERA study for SCA3 (an anti-sense oligonucleotide therapy, see attached letter from the company), but it's important to discontinue trials if they are not safe or helpful so there can be movement and attention towards more safe and effective  therapies. Also, even though the therapy that eventually works for SCA6 may need to be targeted specifically for SCA6, if a particular therapeutic strategy is successful for another SCA, it might motivate the companies to move forward with developing a trial for SCA6 too. 

Best wishes,
Terry

 

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