Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy

• Main Authors: Mirko Signorelli (Author), Burcu Ayoglu (Author), Camilla Johansson (Author), Hanns Lochmüller (Author), Volker Straub (Author), Francesco Muntoni (Author), Erik Niks (Author), Roula Tsonaka (Author), Anja Persson (Author), Annemieke Aartsma‐Rus (Author), Peter Nilsson (Author), Cristina Al‐Khalili Szigyarto (Author), Pietro Spitali (Author)
• Format: Book
• Published: Wiley, 2020-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Duchenne muscular dystrophy (DMD) is a fatal disease for which no cure is available. Clinical trials have shown to be largely underpowered due to inter‐individual variability and noisy outcome measures. The availability of biomarkers able to anticipate clinical benefit is highly...