TAY-SACHS DISEASE
Clinical trials for TAY-SACHS DISEASE explained in plain language.
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New cell booster may help cord blood transplants reach the brain in kids with rare diseases
Disease control Recruiting nowThis early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection.…
Matched conditions: TAY-SACHS DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated May 17, 2026 06:19 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: TAY-SACHS DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC
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New study tracks rare brain diseases to pave way for future cures
Knowledge-focused Recruiting nowThis study follows 52 people with Tay-Sachs, Sandhoff, or GM1 gangliosidosis to understand how these rare genetic diseases progress. Researchers measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so future…
Matched conditions: TAY-SACHS DISEASE
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 11, 2026 20:39 UTC