Mathematical Disease Progression Modeling in Type 2/3 Spinal Muscular Atrophy
Jacqmin P, Gieschke R, Delor I, Snoeck E, Vianna E, Vuillerot C, Sanwald Ducray P.
Muscle Nerve. 2018. 58(4):528-535.
Presentation of a mathematical model that empirically describes the development and deterioration of muscle function assessed by the 3 domains of MFM in Spinal Muscular Atrophy (SMA).
Keywords : Disease progression, Drug development, Mathematical modeling, MFM, Spinal Muscular Atrophy
Articles in scientific journals
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Nagy S, Schmidt S, Hafner P, Klein A, Rubino-Nacht D, Gocheva V, Bieri O, Vuillerot C, Bonati U, Fischer D. J Vis Exp. 2019. (143), e58784. Discussion in a video format on outcome measures, including MFM, used in the trial "Treatment with L-citrulline and metformin in...
Analysis of motor and respiratory function in Duchenne muscular dystrophy patients
Luiz LC, Lima Marson FA, Almeida CCB, Contrera Toro AAD, Nucci A, Ribeiro JD. Respir Physiol Neurobiol. 2019. 262:1-11. Assessment of motor and respiratory impairment of 19 Duchenne muscular dystrophy (DMD) patients. Non-ambulatory condition was associated with worse...
Assessment of disease progression in dysferlinopathy: A 1-year cohort study
Moore U, Jacobs M, James MK, Mayhew AG, Fernandez-Torron R, Feng J, Cnaan A, Eagle M, Bettinson K, Rufibach LE, Lofra RM, Blamire AM, Carlier PG, Mittal P, Lowes LP, Alfano L, Rose K, Duong T, Berry KM, Montiel-Morillo E, Pedrosa-Hernández I, Holsten S, Sanjak M,...