HOW CAN A POSSIBLE TREATMENT APPLY TO OTHER RARE DISEASES?
Rare diseases are surprisingly common! 1 in 10 Americans is affected by a rare disease, with 80% of them involving a problematic gene. The model that Cure CMT4J is pursuing was recently used to treat Batten Disease and Giant Axonal Neuropathy—also monogenic diseases. It represents a pathway to clinical trials that could possibly help the more than 7000 rare and ultra-rare diseases. Cure CMT4J represents Hope for so many other families.

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