Alexander disease
MONDO:0008752Alexander disease (AxD) is a rare neurodegenerative disorder of the astrocytes comprised of two clinical forms: AxD Type I and Type II manifesting with various degrees of macrocephaly, spasticity, ataxia and seizures and leading to psychomotor regression and death.
Also known as: Alexander disease, AxD, ALXDRD, alexanders leukodystrophy, megalencephaly in infancy accompanied by progressive spasticity and dementia
5 clinical trials for this condition and its sub-types, 5 tagged with Alexander disease itself.
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Sub-types of Alexander disease
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Alexander disease type I 0 trials
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Alexander disease type II 0 trials
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New hope for rare brain disease: expanded access to experimental drug zilganersen
Disease control Expanded accessThis program offers early access to zilganersen for people with Alexander disease, a rare genetic brain disorder. It is for US residents aged 2 and older who have not responded to or cannot tolerate approved treatments. The goal is to provide the drug while it is still being stud…
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Major study tracks rare brain disease to pave way for future treatments
Knowledge-focused Recruiting nowThis study follows 200 people with Alexander Disease, a rare brain disorder, to learn how the disease changes over time. Researchers measure movement, speech, swallowing, and quality of life, and collect blood and spinal fluid samples. The goal is to gather information that will …
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples (like blood or tissue) from up to 12,000 people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this data to find new genetic causes, develop biomarkers for future trial…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC