LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.
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Massive newborn study hunts for hidden rare diseases
Diagnosis Recruiting nowThis study aims to screen about 100,000 newborns in the Normandy region of France for two rare genetic disorders called MPS I and Pompe disease. Researchers will use a simple blood spot test collected shortly after birth to see how common these conditions are in the population. T…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase: NA • Sponsor: University Hospital, Rouen • Aim: Diagnosis
Last updated Apr 03, 2026 21:11 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC