LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.
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Mass screening of 100,000 babies aims to catch rare diseases at birth
Diagnosis Recruiting nowThis study aims to screen 100,000 newborns in France for two rare genetic disorders (MPS I and Pompe disease) using a simple blood spot test. The goal is to understand how common these diseases are and to detect them early, when treatment may be most effective. Participation is v…
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase: NA • Sponsor: University Hospital, Rouen • Aim: Diagnosis
Last updated Apr 01, 2026 14:42 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