MLD
Clinical trials for MLD explained in plain language.
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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: MLD
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: MLD
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC