Organized by lgmd2l-foundation
Help us cure a rare and devastating muscle disease, LGMD2L
LGMD2L is a devastating rare disease that destroys muscle. No treatments exist. At the LGMD2L Foundation we have a plan to change that.
With our partner Cure Rare Disease, we have started working on a gene therapy. This therapy will be a one-time infusion that replaces the damaged gene, allowing sufferers a chance of a normal life instead of slowly losing the strength to walk and care for themselves.
With your help, we already raised $25,000 that has paid to develop tools needed to develop and test this gene therapy. That work is underway now at Virginia Commonwealth University. Then we raised $100,000 to develop the actual treatment candidate. Now we're asking for your support to help cover the last milestone before human trials, the studies on its safety and effectiveness.
Both the LGMD2L Foundation and CRD are nonprofits, and 100% of donations will go to pay for the costs of developing the treatment.
Read more here: https://www.businesswire.com/news/home/20260302184269/en/Cure-Rare-Disease-and-LGMD2L-Foundation-Announce-Multi-Year-Partnership-to-Develop-Gene-Therapy-for-Anoctamin-5-Related-Disease
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