Fight CMD was founded by families who refused to accept that nothing could be done for Congenital Muscular Dystrophy. We are dedicated to accelerating research and developing life-changing treatments for children affected by CMD.
Through partnerships with leading researchers and institutions, we're making real progress toward our goal: a treatment for CMD that will transform the lives of affected children and their families.
Fight CMD was founded by families who refused to accept that nothing could be done for Congenital Muscular Dystrophy. We are dedicated to accelerating research and developing life-changing treatments for children affected by CMD.
Through partnerships with leading researchers and institutions, we're making real progress toward our goal: a treatment for CMD that will transform the lives of affected children and their families.
Our drug development program is focused on gene therapy approaches for B3GALNT2-Related Congenital Muscular Dystrophy. The work required to develop a treatment is expensive. Learn about each phase and the costs below.
In this foundational stage, leading researchers develop treatment prototypes to target the genetic root cause of B3GALNT2-Related CMD.
This stage involves rigorous testing in cellular and animal models to determine the optimal dosage and measure safety of a potential therapeutic.
Next, a successful treatment prototype must be "scaled up" into a medical-grade product. Gene therapy requires highly specialized facilities to produce "batches" of the treatment that are pure, stable, and consistent.
This is the final hurdle where the treatment is administered to patients under strict medical supervision. Divided into multiple sub-phases, these trials evaluate how the human body responds to the therapy.
Your donation directly funds critical research and brings us closer to a treatment for CMD.
Join our community, attend events, and help spread awareness about FightCMD.
FightCMD, a non-profit organization (Tax ID number 41-2756694), is qualified as a tax-exempt organization under section 501(c)(3) of the IRS and has been designated as a “public charity” under section 170.
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