ALPHA-MANNOSIDOSIS
Clinical trials for ALPHA-MANNOSIDOSIS explained in plain language.
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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: ALPHA-MANNOSIDOSIS
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Jun 26, 2026 14:27 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: ALPHA-MANNOSIDOSIS
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC