Autosomal recessive Stargardt disease 1 (ABCA4-related)
Clinical trials for Autosomal recessive Stargardt disease 1 (ABCA4-related) explained in plain language.
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Modified vitamin a pill tested against inherited blindness
Disease control OngoingResearchers are testing whether a daily pill called ALK-001 can slow the progression of Stargardt disease, a rare inherited condition that causes vision loss in children and young adults. The trial enrolls people aged 8 and older who have a clinical diagnosis of Stargardt disease…
Matched conditions: AUTOSOMAL RECESSIVE STARGARDT DISEASE 1 (ABCA4-RELATED)
Phase 2 • Sponsor: Alkeus Pharmaceuticals, Inc. • Aim: Disease control
Last updated Sep 21, 2026 22:00 UTC
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Vitamin a derivative eyed to halt inherited blindness
Disease control OngoingResearchers are testing a daily oral drug called ALK-001 in people aged 8 to 70 with Stargardt disease, an inherited condition that causes progressive vision loss. The trial compares ALK-001 to a placebo over 24 months to see if the drug is safe and can slow damage to the retina.…
Matched conditions: AUTOSOMAL RECESSIVE STARGARDT DISEASE 1 (ABCA4-RELATED)
Phase 2 • Sponsor: Alkeus Pharmaceuticals, Inc. • Aim: Disease control
Last updated Sep 21, 2026 22:00 UTC