LYSOSOMAL STORAGE DISEASES
Clinical trials for LYSOSOMAL STORAGE DISEASES explained in plain language.
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Massive newborn screening aims to catch rare diseases early
Diagnosis Recruiting nowThis study will test 100,000 newborns in Normandy, France, for two rare genetic diseases (MPS1 and Pompe disease) using a blood spot test. The goal is to see how common these diseases are and to improve early detection. Parents must give permission for their baby to join.
Matched conditions: LYSOSOMAL STORAGE DISEASES
Phase: NA • Sponsor: University Hospital, Rouen • Aim: Diagnosis
Last updated May 07, 2026 18:43 UTC
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Major study tracks rare brain disorders to unlock disease secrets
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain diseases to learn how these conditions progress. Researchers will track thinking, movement, and daily living skills over time. The goal is to better understand the diseases and how treatments might affect them.
Matched conditions: LYSOSOMAL STORAGE DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC