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NKH Crusaders’ FDA Patient Listening Session: A Milestone in Rare Disease Advocacy
The difficult and often misdirected path to diagnosis
The daily realities of managing severe symptoms like intractable seizures, low muscle tone, developmental delays, and feeding challenges
The emotional and physical toll NKH takes on the entire family
The urgent need for safer, more effective treatments and the willingness of our community to participate in clinical trials
Aug 83 min read


NKH Research Publications
List of known NKH Research Publications
Apr 71 min read
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