Comparative Characterization of a Proposed Generic Nusinersen: Identity of the Oligonucleotide Structure and Equivalence in SMN2 Splicing Activity. [PDF]
PharmaceuticsTaran S +4 more
europepmc +1 more source
Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study. [PDF]
Neurol TherChovi-Trull M +8 more
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MyomiR Networks in Spinal Muscular Atrophy: Associations With Clinical Severity and Treatment Response. [PDF]
Mol NeurobiolBarbo M +4 more
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Treatment of risdiplam after nusinersen continuously improves upper limb motor function in spinal muscular atrophy patients: a multicenter experience. [PDF]
Front PediatrCheng X +6 more
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Rare SMA Patients: A Comprehensive Look at Clinical Features, Genetic Profiles and Therapeutic Approaches. [PDF]
Int J Mol SciMikhalchuk K +14 more
europepmc +1 more source
Enhanced splicing modulation by NMA-modified antisense oligonucleotides. [PDF]
Nucleic Acids ResLing K +14 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
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