Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

Expanded access Protocol to treat LHON subjects with EPI743

Emergency use administration of EPI-743 to subjects with LHON. This Protocol was originally developed to treat a single subject but was expanded to treat several at a single institution.

Inclusion Criteria:

• Emergency treatment for subjects with LHON who are actively losing site in one eye

Exclusion Criteria:

• 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
• Anemia with a HCT <25