Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies

• Main Authors: Pietro Spitali (Author), Kristina Hettne (Author), Roula Tsonaka (Author), Mohammed Charrout (Author), Janneke van denBergen (Author), Zaïda Koeks (Author), Hermien E. Kan (Author), Melissa T. Hooijmans (Author), Andreas Roos (Author), Volker Straub (Author), Francesco Muntoni (Author), Cristina Al‐Khalili‐Szigyarto (Author), Marleen J.A. Koel‐Simmelink (Author), Charlotte E. Teunissen (Author), Hanns Lochmüller (Author), Erik H. Niks (Author), Annemieke Aartsma‐Rus (Author)
• Format: Book
• Published: Wiley, 2018-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Analysis of muscle biopsies allowed to characterize the pathophysiological changes of Duchenne and Becker muscular dystrophies (D/BMD) leading to the clinical phenotype. Muscle tissue is often investigated during interventional dose finding studies to show in situ proof of concep...