SANDHOFF DISEASE
Clinical trials for SANDHOFF 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: SANDHOFF DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated May 26, 2026 07:33 UTC
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Scientists watch rare brain diseases unfold in 1,500 patients
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…
Matched conditions: SANDHOFF DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 26, 2026 07:27 UTC
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Scientists map rare brain diseases to speed future cures
Knowledge-focused Recruiting nowThis study follows 52 people with Tay-Sachs, Sandhoff, or GM1 gangliosidosis to learn how these rare genetic diseases progress. Researchers measure changes in development, movement, and communication skills each year. No new treatments are given; the goal is to create a clear pic…
Matched conditions: SANDHOFF DISEASE
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 26, 2026 07:22 UTC