Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

M. Proud, Crystal; Mercuri, Eugenio; S. Finkel, Richard; Kirschner, Janbernd; C. De Vivo, Darryl; Muntoni, Francesco; Saito, Kayoko; F. Tizzano, Eduardo; Desguerre, Isabelle; Quijano‐Roy, Susana; Benguerba, Kamal; Raju, Dheeraj; Faulkner, Eric; Servais, Laurent

doi:10.1002/acn3.51889

Public

Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

Published in Annals of Clinical and Translational Neurology • Sep 10, 2023

Authors:

Crystal M. Proud

Eugenio Mercuri

Richard S. Finkel

Abstract

We sought to devise a rational, systematic approach for defining/grouping survival motor neuron-targeted disease-modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two-part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Treat...

Subject

Medicine

SMA*

Spinal muscular atrophy

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