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Lebers hereditory optic neuropathy with extra ocular symptoms (lhon-plus)

Clinical trials for Lebers hereditory optic neuropathy with extra ocular symptoms (lhon-plus) explained in plain language.

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Experimental drug aims to boost energy in rare genetic disorders

Disease control Not yet recruiting

This study tests an oral drug called glycerol tributyrate in 24 adults with MELAS or LHON-Plus, two rare mitochondrial diseases that cause severe symptoms like strokes and vision loss. The trial is open-label (everyone gets the drug) and uses each person as their own control over…

Matched conditions: LEBERS HEREDITORY OPTIC NEUROPATHY WITH EXTRA OCULAR SYMPTOMS (LHON-PLUS)

Phase 1/2 • Sponsor: George Washington University • Aim: Disease control

Last updated Jun 27, 2026 13:02 UTC

Lebers hereditory optic neuropathy with extra ocular symptoms (lhon-plus)

Get alerted when new Lebers hereditory optic neuropathy with extra ocular symptoms (lhon-plus) trials appear

Experimental drug aims to boost energy in rare genetic disorders