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Nusinersen Wearing-Off in Adult 5q-Spinal Muscular Atrophy Patients

The antisense oligonucleotide nusinersen was the first drug treatment available for all types of 5q-spinal muscular atrophy (SMA). The dosing regime has been derived from pivotal clinical trials in infants and children. The efficacy of nusinersen in severely affected adult SMA patients is still ques...

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Bibliografiset tiedot
Päätekijät: Alma Osmanovic, Olivia Schreiber-Katz, Susanne Petri
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: MDPI AG 2021-03-01
Sarja:Brain Sciences
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Linkit:https://www.mdpi.com/2076-3425/11/3/367
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