SANDHOFF DISEASE
Clinical trials for SANDHOFF DISEASE explained in plain language.
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Promising new pill for rare childhood brain diseases enters final testing
Disease control Recruiting nowThis study tests an oral drug called nizubaglustat in children and teens with rare genetic disorders (GM1, GM2 gangliosidosis) that damage the brain and nerves. The goal is to see if the drug can slow disease progression and improve movement and coordination. About 75 participant…
Matched conditions: SANDHOFF DISEASE
Phase: PHASE3 • Sponsor: Azafaros B.V. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases
Disease control Recruiting nowThis early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…
Matched conditions: SANDHOFF DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Jun 26, 2026 14:27 UTC
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Scientists track rare brain diseases to pave way for future cures
Knowledge-focused Recruiting nowThis study follows children and adults with Tay-Sachs, Sandhoff, and GM1 gangliosidosis to understand how these diseases progress. Researchers will measure changes in motor skills, communication, and behavior each year. The goal is to create a clear picture of the diseases so fut…
Matched conditions: SANDHOFF DISEASE
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC
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Major study tracks rare brain diseases to unlock their secrets
Knowledge-focused Recruiting nowThis study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how tr…
Matched conditions: SANDHOFF DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC