Rare diseases affect 1 in 10 people.
Finding the cure for one can benefit thousands.
Donate Today
Rare diseases affect 1 in 10 people.
Finding the cure for one can benefit thousands.
Donate Today
Focused Research Using Gene Therapy

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CMT4J is a rare, progressive neurological disease quite similar to ALS. People with CMT4J experience progressive muscle weakness, leading to difficulty walking or wheelchair dependence.

Like many neuromuscular diseases, the disease can progress to involve respiratory function, resulting in pneumonias, respiratory failure, and untimely death.

CureCMT4J is working to advance the science toward a clinical trial using gene therapy to treat or cure CMT4J. Our pre-clinical work shows early, very promising results in mice.

Although CMT4J is an ultra-rare disease, the science we're working on can be translated to thousands of other rare diseases.
THE TIME IS NOW. THE FUNDING IS CRUCIAL TO MOVE FORWARD.

Rare diseases like CMT4J need your help to fund a cure.

Because they affect so few, they receive little funding for research—even though advancing the research for one rare disease can have a big impact on advancing treatment for all rare diseases.

You can make a difference. Donate Today.

Funding raised to date:
$447,370
OF OUR $1,000,000 GOAL
Donate Today

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