Results 41 to 50 of about 6,290 (252)
Journal of Market Access & Health Policy, 2021 Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results.
Rebecca Dean +11 more
doaj +1 more source
The effects of nusinersen treatment on respiratory status of children with spinal muscular atrophy [PDF]
, 2022 BACKGROUND: Respiratory involvement is the main factor predicting the prognosis of spinal muscular atrophy (SMA). Significant responses in motor functions have been demonstrated with nusinersen, but pulmonary outcomes are still varied.
Arslan, Hüseyin +9 more
core +1 more source
BMJ Paediatrics Open, 2019 Background Nusinersen has been used to treat spinal muscular atrophy type 1 (SMA1) in the UK since 2017. While initial trials showed neuromuscular benefit from treating SMA1, there is little information on the respiratory effects of nusinersen.
Francis J Gilchrist +7 more
doaj +1 more source
Is cerebrospinal fluid amyloid-β42 a promising biomarker of response to nusinersen in adult spinal muscular atrophy patients? [PDF]
, 2021 Introduction: Nusinersen was approved as the first treatment for all types of spinal muscular atrophy (SMA), including adults with SMA types 2 and 3. Robust biomarkers of treatment response in SMA adults are lacking.
D'Errico E. +7 more
core +1 more source
Spinal muscular atrophy 5Q – Treatment with nusinersen [PDF]
Revista da Associação Médica Brasileira, 2018 The Guidelines Project, an initiative of the Brazilian Medical Association, aims to combine information from the medical field in order to standardize producers to assist the reasoning and decision-making of doctors. The information provided through this project must be assessed and criticized by the physician responsible for the conduct that will be ...
Antonio Silvinato, Wanderley M Bernardo
openaire +5 more sources
Bridging Clinical Outcomes and Cost-Effectiveness: The Role of Real-World Data in Cystic Fibrosis Therapy. [PDF]
Pharmacol Res PerspectPharmacology Research &Perspectives, Volume 13, Issue 4, August 2025.
Štimac I +3 more
europepmc +2 more sources
Antisense oligonucleotides and other genetic therapies made simple [PDF]
, 2018 Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors.
Reilly, M, Rossor, A, Sleigh, JN
core +1 more source
Comparison of Nusinersen and Evrysdi in the Treatment of Spinal Muscular Atrophy [PDF]
E3S Web of Conferences, 2021 Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease that commonly affects children, and usually worsens with age that often leads to permanent disability and death for many of the SMA patients.
Zhu Xiaoying
doaj +1 more source
Gene suppression approaches to neurodegeneration [PDF]
, 2017 Gene suppression approaches have emerged over the last 20 years as a novel therapeutic approach for the treatment of neurodegenerative diseases. These include RNA interference and anti-sense oligonucleotides, both of which act at the post-transcriptional
Ghosh, R, Tabrizi, SJ
core +1 more source
Elderly patient with 5q spinal muscular atrophy type 4 markedly improved by Nusinersen [PDF]
, 2020 Available online 17 May 2020.ArticleJournal of the Neurological Sciences.415:116901(2020)journal ...
Morizumi, Teruya +6 more
core +1 more source