Alpha-mannosidosis
MONDO:0009561Alpha-mannosidosis is an inherited lysosomal storage disorder characterized by immune deficiency, facial and skeletal abnormalities, hearing impairment, and intellectual deficit.
Also known as: alpha-mannosidosis, lysosomal alpha-D-mannosidase deficiency, mannosidosis, alpha-, types I and II, Alpha mannosidase B deficiency, Alpha-mannosidase B deficiency, MANSA, lysosomal Alpha-D-mannosidase deficiency, mannosidosis, ALPHA B, lysosomal
5 clinical trials for this condition and its sub-types, 5 tagged with Alpha-mannosidosis itself.
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Sub-types of Alpha-mannosidosis
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Alpha-mannosidosis type 1 0 trials
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Alpha-mannosidosis, adult form 0 trials
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Alpha-mannosidosis, infantile form 0 trials
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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…
Phase 1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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Milder chemo before stem cell transplant shows promise for rare blood disorders
Disease control Recruiting nowThis study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…
Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Milder transplant method aims to help young patients with rare blood diseases
Disease control Recruiting nowThis study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…
Phase 2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Jun 27, 2026 08:04 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…
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:03 UTC