GAA DEFICIENCY

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Tracking a rare disease from birth: study seeks clues for better future care

Knowledge-focused Ongoing

This study aims to learn more about the early signs and symptoms of late-onset Pompe disease in babies and young children who are diagnosed through newborn screening. Researchers will closely monitor 20 children over several years to understand how the disease develops and to hel…

Matched conditions: GAA DEFICIENCY

Sponsor: Duke University • Aim: Knowledge-focused

Last updated Mar 30, 2026 14:29 UTC

GAA DEFICIENCY

Get alerted when new GAA DEFICIENCY trials appear

Tracking a rare disease from birth: study seeks clues for better future care