ADRENOLEUKODYSTROPHY
Clinical trials for ADRENOLEUKODYSTROPHY explained in plain language.
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New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression
Disease control Recruiting nowThis early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly i…
Matched conditions: ADRENOLEUKODYSTROPHY
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Massive global hunt for answers to devastating brain diseases
Knowledge-focused Recruiting nowThis study aims to collect medical information and biological samples from 12,000 people worldwide with rare genetic brain disorders called leukodystrophies. Researchers will use this data to better understand these diseases, improve diagnosis, and identify targets for future tre…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC
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Scientists probe immune link to devastating brain disorders
Knowledge-focused Recruiting nowThis study aims to understand how the body's immune cells, specifically macrophages, might influence the progression of several rare inherited brain diseases. Researchers will collect a single blood sample from 100 participants, including patients with conditions like adrenoleuko…
Matched conditions: ADRENOLEUKODYSTROPHY
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 20, 2026 14:48 UTC