CMT4J RESEARCH PATHWAYS and PARTNERS in the news:
1. FIG4/AAV9 GENE THERAPY
Together with our partner, ELPIDA THERAPEUTICS, WE ARE THRILLED TO ANNOUNCE OUR UPCOMING GENE THERAPY CLINICAL TRIAL, SET TO BEGIN IN SPRING/SUMMER, 2026!
VISIT THE NIH CLINICAL TRIALS WEBSITE TO LEARN MORE
HOW DOES GENE THERAPY WORK? A benign virus, called AAV9 carries healthy copies of the FIG4 gene, which is malfunctioning and under-functioning in people with CMT4J. The hope is that the trillions of new copies of the FIG4 gene will take over and stop progression of CMT4J, providing the potential for lifesaving, life-changing intervention.
2. CALIFORNIA INSTITUTES FOR REGENERATIVE MEDICINE (CIRM) AWARDS ELPIDA THERAPEUTICS AN $8MM GRANT TO BEGIN GENE THERAPY CLINICAL TRIALS IN CMT4J!
3.FDA DESIGNATES CMT4J AS RARE PEDIATRIC DISEASE. AWARDS ORPHAN DRUG DESIGNATION AND RARE PEDIATRIC DISEASE DESIGNATION, PROVIDING ACCELERATED PATHWAYS TOWARD AAV9/FIG4 GENE THERAPY CLINICAL TRIALS AND FUTURE APPROVAL
4. CMTRF JOINS CURECMT4J AND ELPIDA THERAPEUTICS IN FUNDING THE MANUFACTURING OF OUR /FIG4 GENE THERAPY!
READ MORE HERE
5. DRUG REPURPOSING
Through research known as “high throughput screening” CureCMT4J has identified several potential drugs already FDA-approved that might also be beneficial to CMT4J patients. We continue to try to bring these discoveries out of the lab and into the patients.
THANK YOU!
Every single day, we continue to research, identify, and push forward potential treatments for individuals with CMT4J. We’ve learned over the past few years that, unfortunately, it isn’t a sprint to the finish line. It is a marathon. It is a slog. Science is rarely straightforward. Regulatory roadblocks grow larger every year. Research funding and manufacturing delays still represent the biggest hurdles for ultra-rare diseases. But our work won’t stop. We hope you’ll continue to support us!
DOES YOUR CHILD OR DO YOU HAVE CMT4J? WANT TO LEARN MORE ABOUT OUR WORK OR HOW YOU CAN HELP US TO RAISE FUNDS TOWARD THE RESEARCH? CONTACT US: jocelyn@curecmt4j.org
We need you!
We always need help! If you believe you have expertise and interest in any of the following areas, we’d love to hear from you:
Research/Science – Do you have experience in medicine, research, bioinformatics, or epidemiology? Can you read and interpret scientific studies?
Web and Content Design/Tech Support – Do you have experience in designing websites? Hosting virtual international conferences?
Contact us here: jocelyn@curecmt4j.org
Fundraising– Want to host a fundraiser for CureCMT4J? Go to our website for some ideas: http://www.curecmt4j.org/get-involved/.
SHOELACES!
We have lots of CureCMT4J shoelaces if you’d like to use them in your fundraiser! (suggested donation $5/pair [individual non-event lace orders = $5/pair + shipping) Contact us at: jocelynaduff@gmail.com
FACEBOOK BIRTHDAY FUNDRAISERS!
Want a simple way to fundraise for and spread the word about CureCMT4J ? If you have a Facebook account you can host a Birthday Fundraiser every year on your special day! Go here to find out how to do it: https://www.facebook.com/help/1910205189301966/ [Facebook receives no money raised in these online FB fundraisers]
Or DONATE on a one-time or monthly basis on our website!
Thank you!!!
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