<?xml version="1.0" encoding="UTF-8"?><xml><records><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>17</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Bram De Wel</style></author><author><style face="normal" font="default" size="100%">Veerle Goosens</style></author><author><style face="normal" font="default" size="100%">Atka Sobota</style></author><author><style face="normal" font="default" size="100%">Elke Van Camp</style></author><author><style face="normal" font="default" size="100%">Ellen Geukens</style></author><author><style face="normal" font="default" size="100%">Griet Van Kerschaver</style></author><author><style face="normal" font="default" size="100%">Marlène Jagut</style></author><author><style face="normal" font="default" size="100%">Kathleen Claes</style></author><author><style face="normal" font="default" size="100%">Kristl G. Claeys</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4</style></title><secondary-title><style face="normal" font="default" size="100%">Journal of Neurology</style></secondary-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">SMA · Spinraza® · Muscle strength · ActivLim · Outcome measures · Natural history</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2020</style></year><pub-dates><date><style  face="normal" font="default" size="100%">Mar-09-2021</style></date></pub-dates></dates><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;Abstract&lt;br&gt;
Background Nusinersen recently became available as the first treatment for Spinal Muscular Atrophy (SMA) and data on&lt;br&gt;
its effectiveness and safety in adult SMA patients are still scarce.&lt;br&gt;
Methods We evaluated the effectiveness and safety of nusinersen treatment during 14 months in 16 adult patients with SMA&lt;br&gt;
types 3 and 4 in a prospective study, and retrospectively detailed the natural history of 48 adult SMA patients types 2, 3 and 4.&lt;br&gt;
Results Hand grip strength (p = 0.03), hand motor function (p = 0.04) as assessed by a sub-score of the Revised Upper&lt;br&gt;
Limb Module (RULM) and the Medical Research Council (MRC) sum score (p = 0.04) improved significantly at month 14.&lt;br&gt;
Importantly, the MRC sum score had declined significantly (p &amp;lt; 0.01) prior to start of treatment in these patients. A minimal&lt;br&gt;
clinically important difference (MCID) in the Hammersmith Functional Motor Scale Expanded (HFMSE) and RULM scores&lt;br&gt;
was achieved in 31% and 50% of the patients, respectively, but the mean changes from baseline failed to reach significance.&lt;br&gt;
Forced Vital Capacity (FVC) transiently increased at month 6 (p = 0.01), whereas the Peak Expiratory Flow (PEF) did not.&lt;br&gt;
The Activity Limitations scale declined significantly prior to start of treatment (p &amp;lt; 0.01) and showed an improvement with&lt;br&gt;
nusinersen which was not significant. The safety evaluation did not reveal serious adverse events and no signs of nephrotoxicity&lt;br&gt;
or antisense oligonucleotide (ASO)-mediated inflammation.&lt;br&gt;
Conclusions We conclude that hand grip strength and hand motor function, as well as MRC sum scores improved significantly&lt;br&gt;
in nusinersen-treated adult patients with SMA types 3 and 4.&lt;/p&gt;
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