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.
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.
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