ALEXANDER DISEASE
Clinical trials for ALEXANDER DISEASE explained in plain language.
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Rare brain disease patients get early access to experimental drug
Disease control AVAILABLEThis program offers zilganersen to people with Alexander disease, a rare genetic brain disorder, who have not responded to or cannot tolerate approved treatments. It is for US residents aged 2 and older with a confirmed GFAP gene variant. The goal is to provide access to this exp…
Matched conditions: ALEXANDER DISEASE
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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Massive leukodystrophy biobank aims to unlock disease secrets
Knowledge-focused Recruiting nowThis study collects medical information and biological samples from up to 12,000 people with leukodystrophies—rare brain disorders affecting white matter. Researchers will use this data to find new genetic causes, develop biomarkers, and better understand how these diseases progr…
Matched conditions: ALEXANDER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 13, 2026 16:02 UTC
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New study tracks alexander disease to pave way for future treatments
Knowledge-focused Recruiting nowThis study follows 200 people with Alexander Disease, a rare brain disorder, to see how symptoms like movement, speech, and swallowing change over time. Researchers collect samples and test results to better understand the disease. The information gathered will help design future…
Matched conditions: ALEXANDER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 07, 2026 18:42 UTC