Compilation of online resources of relevance to 'Spinraza and advanced therapies:a stakeholder special' issue of Gene Therapy [PDF]
, 2017 Prakash, V, Yáñez-Muñoz, R J
core +1 more source
Why antisense could make sense for neurodegeneration [PDF]
, 2017 Massey, Thomas, Robertson, Neil
core +1 more source
Protein production is an early biomarker for RNA‐targeted therapies [PDF]
, 2018 Alex, Jacob +10 more
core +2 more sources
A historical and current review of newborn screening for neuromuscular disorders from around the world: lessons for the United States [PDF]
, 2017 Clarke, Angus John +1 more
core +2 more sources
Gene Therapy, more than ever-a new vision for the journal [PDF]
, 2017 Yáñez-Muñoz, R J
core +1 more source
“Not Only Has She Survived, But She Lives a Happy Life”: Parents’ Perspectives and Experiences of a Novel Disease-Modifying Therapy for Spinal Muscular Atrophy in Sweden [PDF]
The objective of this prospective cohort study was to explore parents' perspectives of patients' experiences of the first available novel disease-modifying therapy for SMA in Sweden.
Cortina-Borja, Mario +4 more
core
Development and Validation of an Ion-Pair Reverse-Phase High-Performance Liquid Chromatography-Electrospray Ionization Mass Spectrometry Method for Determination of Purity of Nusinersen for Quality Control of Drug Substance or Drug Product. [PDF]
Int J Mol SciSamoilov M, Zubareva E, Degterev M.
europepmc +1 more source
Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. [PDF]
BMC NeurolYao X +15 more
europepmc +1 more source
Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients. [PDF]
Front PediatrHe G +5 more
europepmc +1 more source
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
europepmc +1 more source