Neurodevelopmental Comorbidities in Early-Onset 5q-SMA Treated with Disease-Modifying Therapies: A Scoping Review [PDF]
Recent studies have reported possible neurodevelopmental comorbidities in some early onset 5q-spinal muscular atrophy (5q-SMA) patients treated with disease-modifying therapies (DMT).
Konop, Carson
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Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]
J NeurolNeuhoff S +11 more
europepmc +1 more source
The effectiveness and value of therapies for spinal muscular atrophy. [PDF]
J Manag Care Spec PharmTice JA +9 more
europepmc +1 more source
Effectiveness of Risdiplam Treatment in Adult Patients With Spinal Muscular Atrophy Type IIb-III. [PDF]
Rev NeurolGarcía Estévez DA.
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Preventing spinal muscular atrophy through the national premarital screening program in Türkiye: an economic comparison with treatment costs. [PDF]
Turk J Med SciDur G, Tecirli G, Okumuş N.
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Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review. [PDF]
J NeurolAlonge P, Urbano G, Gadaleta G.
europepmc +1 more source
Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence. [PDF]
J Int Med ResSel EK +6 more
europepmc +1 more source
Analyse der gesundheitsbezogenen Lebensqualität von Patienten mit spinaler Muskelatrophie (SMA) unter der Therapie mit Nusinersen und Ermittlung der Belastung ihrer pflegenden Angehörigen [PDF]
Velling, Ann-Kathrin
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Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment [PDF]
, 2020 Boido, Marina Maria +3 more
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A Chemically Induced CRISPR/dCas13<sup>FCPF</sup> Platform for Precise and Programmable RNA Regulation. [PDF]
J Med ChemHasselbeck S +6 more
europepmc +1 more source