ALEXANDER DISEASE
Clinical trials for ALEXANDER DISEASE explained in plain language.
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New hope for rare brain disease: expanded access program opens
Disease control AVAILABLEThis program offers zilganersen to people with Alexander disease, a rare genetic condition that damages the nervous system. It is for US residents aged 2 and older who have not responded to or cannot tolerate other treatments. The goal is to provide access to this experimental dr…
Matched conditions: ALEXANDER DISEASE
Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 20, 2026 12:02 UTC
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Massive leukodystrophy biobank aims to unlock secrets of rare brain diseases
Knowledge-focused Recruiting nowThis study is creating a large collection of medical data and biological samples from people with leukodystrophies—rare disorders that damage the brain's white matter. Researchers will use this information to discover new genetic causes, develop better diagnostic tools, and track…
Matched conditions: ALEXANDER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 09:59 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 condition 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 wil…
Matched conditions: ALEXANDER DISEASE
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 26, 2026 09:58 UTC