Results from a 3-year Non-interventional, Observational Disease Monitoring Program in Adults with GNE Myopathy.
Hanns Lochmüller,
Anthony Behin,
Ivailo Tournev,
Mark Tarnopolsky,
Rita Horváth,
Oksana Pogoryelova,
Jinay Shah,
Tony Koutsoukos,
Alison Skrinar,
Emil Kakkis,
Camille L Bedrosian,
Tahseen Mozaffar
Mar 30, 2021
BACKGROUND: GNE myopathy is a rare, autosomal recessive, muscle disease caused by mutations in GNE and is characterized by rimmed vacuoles on muscle biopsy and progressive distal to proximal muscle weakness. OBJECTIVE...