Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]
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Layout table for eligibility information
Ages Eligible for Study:
8 Years to 65 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:
Emergency treatment for subjects with LHON who are actively losing site in one eye
Allergy to EPI-743 or sesame oil
Clinical history of bleeding or abnormal PT/PTT
Hepatic insufficiency with LFTs greater than 2-times normal
Renal insufficiency requiring dialysis
Fat malabsorption syndromes
Any other concurrent inborn erros of metabolism
Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis