Assessment of Health-Related Quality of Life in Adult Spinal Muscular Atrophy Under Nusinersen Treatment—A Pilot Study

Assessment of Health-Related Quality of Life in Adult Spinal Muscular Atrophy Under Nusinersen Treatment—A Pilot Study

5q-Spinal muscular atrophy (SMA) is a severely disabling inherited neuromuscular disease that progressively reduces the motor abilities of affected individuals. The approval of the antisense oligonucleotide nusinersen, which has been shown to improve motor function in adult SMA patients, changed the...

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Bibliographic Details
Main Authors: Andreas Thimm, Svenja Brakemeier, Kathrin Kizina, Juan Munoz Rosales, Benjamin Stolte, Andreas Totzeck, Cornelius Deuschl, Christoph Kleinschnitz, Tim Hagenacker
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-01-01
Series:Frontiers in Neurology
Subjects:
spinal muscular atrophy
nusinersen
Neuro-QoL
antisense oligonucleotide
HFMSE
RULM
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2021.812063/full

Internet

https://www.frontiersin.org/articles/10.3389/fneur.2021.812063/full

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