Cerebral adrenoleukodystrophy (CALD)
Clinical trials for Cerebral adrenoleukodystrophy (CALD) explained in plain language.
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One-Time gene therapy for fatal childhood brain disease: does it last?
Cure OngoingResearchers are following 64 people with cerebral adrenoleukodystrophy (CALD) for 15 years after they received a one-time gene therapy called eli-cel in earlier studies. The therapy uses the patient's own stem cells, modified to carry a working copy of the gene that is faulty in …
Matched conditions: CEREBRAL ADRENOLEUKODYSTROPHY (CALD)
Sponsor: Genetix Biotherapeutics Inc. • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC
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Hope for rare brain disease: new drug trial aims to stall deadly decline
Disease control OngoingThis Phase 3 study tests whether the drug leriglitazone can help adult men with cerebral adrenoleukodystrophy (cALD), a rare and serious brain disease. The main goal is to see if the drug delays death or the need for permanent breathing support compared to a placebo. About 40 men…
Matched conditions: CEREBRAL ADRENOLEUKODYSTROPHY (CALD)
Phase 3 • Sponsor: Minoryx Therapeutics, S.L. • Aim: Disease control
Last updated Jul 31, 2026 00:00 UTC