MENKES DISEASE

Clinical trials for MENKES DISEASE explained in plain language.

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Trials to join now! 1 Not yet finished but already full! 2 Completed 1

Drug shows promise for rare disease Survivors' daily struggles

Symptom relief Completed

This study tested whether the drug Northera (droxidopa) is safe and helpful for adults with Menkes disease or Occipital Horn Syndrome who experience low blood pressure and other nervous system symptoms. Three participants took either the drug or a placebo in a crossover design. T…

Matched conditions: MENKES DISEASE

Phase: PHASE1, PHASE2 • Sponsor: Stephen G. Kaler, MD • Aim: Symptom relief

Last updated Apr 24, 2026 16:20 UTC

MENKES DISEASE

Get alerted when new MENKES DISEASE trials appear

Drug shows promise for rare disease Survivors' daily struggles