Spinal Muscular Atrophy Foundation

Recent papers from Spinal Muscular Atrophy Foundation

1. CCN1-mediated stress signaling underlies intrinsic cardiomyocyte dysfunction in spinal muscular atrophyCell Reports · 2026
2. Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular AtrophyInternational Journal of Molecular Sciences · 2024
3. Targeted Stimulation of the Sensory Afferents Improves Motoneuron Function in Humans With Spinal Muscular Atrophy2024
4. Targeted Stimulation of the Sensory Afferents Improves Motoneuron Function in Humans With a Degenerative Motoneuron Disease2024
5. Identifying Biomarkers of Spinal Muscular Atrophy for Further DevelopmentJournal of Neuromuscular Diseases · 2023
6. Advances and limitations for the treatment of spinal muscular atrophyBMC Pediatrics · 2022
7. Ethical aspects of the changing landscape for spinal muscular atrophy management in AustraliaAustralian Journal of General Practice · 2022
8. Activation of Muscle-Specific Kinase (MuSK) Reduces Neuromuscular Defects in the Delta7 Mouse Model of Spinal Muscular Atrophy (SMA)International Journal of Molecular Sciences · 2021
9. Gastrointestinal bleeding prophylaxis for critically ill patients: a clinical practice guidelineBMJ · 2020
10. Development of the Initial Surveys for the All of Us Research ProgramEpidemiology · 2019
11. SMN Protein Can Be Reliably Measured in Whole Blood with an Electrochemiluminescence (ECL) Immunoassay: Implications for Clinical TrialsSHILAP Revista de lepidopterología · 2016
12. Rasch analysis of clinical outcome measures in spinal muscular atrophyMuscle & Nerve · 2013
13. SMA-MAP: A Plasma Protein Panel for Spinal Muscular AtrophyPLoS ONE · 2013
14. Evaluation of Peripheral Blood Mononuclear Cell Processing and Analysis for Survival Motor Neuron ProteinPLoS ONE · 2012
15. Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical StudyPLoS ONE · 2012
16. Candidate Proteins, Metabolites and Transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical StudyPLoS ONE · 2012
17. Utility of Survival Motor Neuron ELISA for Spinal Muscular Atrophy Clinical and Preclinical AnalysesPLoS ONE · 2011
18. Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophyJournal of Clinical Investigation · 2011