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  • Publication : 28 06 2018
  • Catégorie :La recherche médicale et génétique

Mathematical Disease Progression Modeling in Type 2/3 Spinal Muscular Atrophy.    

Introduction We propose a mathematical model to empirically describe spinal muscular atrophy (SMA) progression assessed by the three domains of the motor function measure (MFM) scale. The model implements development and deterioration of muscle function. Methods Nonlinear mixed-effects modeling was applied to data from two observational and one prospective clinical efficacy study comprising in total 190 healthy subjects and 277 Type 2/3 SMA patients. Results The model evidenced correlations between parameters for different MFM domains. Slower development in MFM domain D1 was associated with faster deterioration for MFM domains D2 and D3. Discussion The model describes all individual data well, although sparseness and variability of observational data prevented numerically stable estimation of parameters. Treatment duration in clinical studies was too limited to determine a proper drug effect model that could differentiate between symptomatic and disease modifying effect. This article is protected by copyright. All rights reserved.

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