TAY-SACHS DISEASE
Clinical trials for TAY-SACHS DISEASE explained in plain language.
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New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression
Disease control Recruiting nowThis early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly i…
Matched conditions: TAY-SACHS DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Scientists map the devastating course of rare childhood diseases
Knowledge-focused Recruiting nowThis study aims to carefully track how rare, inherited disorders called gangliosidoses (like Tay-Sachs and Sandhoff disease) progress over time. Researchers will follow about 52 participants, from infants to adults, using tests and brain scans to measure changes in development, m…
Matched conditions: TAY-SACHS DISEASE
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 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: TAY-SACHS DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC