Menkes Disease

Disease

Disease Profile

Menkes disease is a rare X-linked disorder of copper transport caused by ATP7A variants, resulting in severe neurodevelopmental decline, connective tissue abnormalities, and characteristic hair changes.

Category
Endocrine, nutritional and metabolic diseases
Prevalence
Estimated incidence about 1 in 100,000 newborns
ICD Codes
  • E83.0

Related News

FDA Approves First Generic Glatiramer Acetate and Pediatric Menkes Treatment in Q1 2026

The FDA approved ScinoPharm Taiwan's glatiramer acetate as the first complex injectable generic for relapsing multiple sclerosis and Sentynl Therapeutics' copper histidinate as the first treatment for pediatric Menkes disease in January 2026. These approvals represent significant advancements in neurological care, with the glatiramer acetate approval following a novel regulatory pathway and the Menkes treatment showing improved survival in early-treated patients.

Related Clinical Trials

NCT ID Title Status Phase
NCT04977388

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

COMPLETED PHASE1/PHASE2