ALEXANDER DISEASE
Clinical trials for ALEXANDER DISEASE explained in plain language.
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New hope for rare brain disease: expanded access to experimental drug zilganersen
Disease control AVAILABLEThis 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…
Matched conditions: ALEXANDER DISEASE
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 …
Matched conditions: ALEXANDER DISEASE
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…
Matched conditions: ALEXANDER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC