Breaking News:
4th
ACDase Deficiency Global Forum — Save the date February 17, 2027
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Website Updated: June 2, 2026
Saving children's
lives with
breakthrough research
We are an approved 501(c)(3) nonprofit dedicated to finding solutions for Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy. Researchers are finding solutions — and children are living in hope of a cure.
1–4
new cases diagnosed every month
Based upon birth prevalence studies
Our current focus
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