Muscle MRI: A biomarker of disease severity in Duchenne muscular dystrophy? A systematic review
Ropars J, Gravot F, Ben Salem D, Rousseau F, Brochard S, Pons C.
Neurology. 2020. 94(3):117-133.
Review to assess the evidence of a relationship between muscle MRI and disease severity in Duchenne muscular dystrophy (DMD).
Correlations between motor function tests, including MFM, and MRI measurements are discussed.
Articles in scientific journals
Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy.
Nagy S, Schädelin S, Hafner P, Bonati U, Scherrer D, Ebi S, Schmidt S, Orsini AL, Bieri O, Fischer D. Muscle Nerve. 2020. 61(1), 63-68. In this retrospective study, the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD...
Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies.
Jain MS, Meilleur K, Kim E, Norato G, Waite M, Nelson L, McGuire M, Duong T, Keller K, Lott DJ, Glanzman A, Rose K, Main M, Fiorini C, Chrismer I, Linton M, Punjabi M, Elliott J, Tounkara F, Vasavada R, Logaraj R, Winkert J, Donkervoort S, Leach M, Dastgir J, Hynan L,...
Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial
Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D. JAMA Netw Open. 2019. 2;2(10):e1914171. Results of a single-center randomized...
Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial.
Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schädelin S, Bieri O, Fischer D. Trials. 2019. 20(1), 637. Protocol of a multicenter, randomized, double-blind, placebo-controlled phase III trial study aiming to demonstrate safety and efficacy of tamoxifen over placebo in...
Adult MTM1-related myopathy carriers: Classification based on deep phenotyping
Cocanougher BT, Flynn L, Yun P, Jain M, Waite M, Vasavada R, Wittenbach JD, de Chastonay S, Chhibber S, Innes AM, MacLaren L, Mozaffar T, Arai AE, Donkervoort S, Bönnemann CG, Foley AR. Neurology. 2019. 93 (16), e1535-e1542. Results of a cohort study performed at the...
Clinical trial readiness to solve barriers to drug development in FSHD (ReSolve): protocol of a large, international, multi-center prospective study.
LoRusso S, Johnson NE, McDermott MP, Eichinger K, Butterfield RJ, Carraro E, Higgs K, Lewis L, Mul K, Sacconi S, Sansone VA, Shieh P, van Engelen B, Wagner K, Wang L, Statland JM, Tawil R; ReSolve Investigators and the FSHD CTRN. BMC Neurol. 2019. 10;19(1):224. The...