PELIZAEUS-MERZBACHER DISEASE
Clinical trials for PELIZAEUS-MERZBACHER DISEASE 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: PELIZAEUS-MERZBACHER DISEASE
Phase: PHASE1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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First human trial launches for rare childhood brain disease
Disease control Recruiting nowThis study is the first to test a new drug called ION356 in children with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder that affects the brain and nerves. The main goal is to check if the drug is safe and how the body processes it when given as a spinal injection. A…
Matched conditions: PELIZAEUS-MERZBACHER DISEASE
Phase: PHASE1 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 17, 2026 13:10 UTC
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Scientists track rare brain disease in children to find clues for future treatments
Knowledge-focused Recruiting nowThis study aims to understand how Pelizaeus-Merzbacher disease (PMD) progresses over time in boys aged 6 months to 17 years. Researchers will collect spinal fluid, blood samples, and brain scans to identify biological markers that change as the disease advances. The information g…
Matched conditions: PELIZAEUS-MERZBACHER DISEASE
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Mar 31, 2026 12:10 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: PELIZAEUS-MERZBACHER DISEASE
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 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: PELIZAEUS-MERZBACHER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:09 UTC