X-linked adrenoleukodystrophy (X-ald)

Clinical trials for X-linked adrenoleukodystrophy (X-ald) explained in plain language.

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One-Time gene therapy for fatal childhood brain disease: does it last?

Cure Ongoing

Researchers 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: X-LINKED ADRENOLEUKODYSTROPHY (X-ALD)

Sponsor: Genetix Biotherapeutics Inc. • Aim: Cure

Last updated Sep 16, 2026 00:00 UTC

X-linked adrenoleukodystrophy (X-ald)

Get alerted when new X-linked adrenoleukodystrophy (X-ald) trials appear

One-Time gene therapy for fatal childhood brain disease: does it last?