Cure Spinal Muscular Atrophy

Recent papers from Cure Spinal Muscular Atrophy

1. Defining meaningful treatment outcomes in adults with spinal muscular atrophy: an expert consensus approachFrontiers in Neurology · 2026
2. Trial-related informational needs and decision-making in the SMA community after broad treatment access: results of the 2nd Cure SMA clinical trial experience surveyOrphanet Journal of Rare Diseases · 2026
3. Validation of a tissue-based predictive RNA test for immunotherapy benefit in NSCLC: the PREDAPT studynpj Precision Oncology · 2026
4. Exploration of Patient and Caregiver Burden Due to Insurance Denials and Appeals for Spinal Muscular Atrophy (SMA) Treatment and Healthcare Access (P5-9.004)Neurology · 2026
5. Exploration of the Diagnostic and Treatment Journey of Adult-onset (Type Four) SMA (P5-9.003)Neurology · 2026
6. An updated review of the SMA clinical trial landscape in the United States: Findings from analysis of recruitment targets on ClinicalTrials.gov and a survey of SMA clinical trial sites on factors affecting site capacity and readinessContemporary Clinical Trials Communications · 2026
7. A qualitative, mixed-method approach to reaching consensus on function, fatigue, and fatigability outcomes in teens and adults living with spinal muscular atrophyOrphanet Journal of Rare Diseases · 2025
8. SMA Community Priorities for Future Drug Therapies: Cross-Sectional Survey Findings Representing 410 Adults with SMANeurology and Therapy · 2025
9. Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scaleJournal of Neuromuscular Diseases · 2025
10. Newborn Screening and Birth Prevalence for Spinal Muscular Atrophy in the USJAMA Pediatrics · 2024
11. Spinal Muscular Atrophy Update in Best PracticesNeurology Clinical Practice · 2024
12. Delphi Consensus Panel on Assessing Clinically Meaningful Treatment Outcomes in Adults Living with Spinal Muscular Atrophy (SMA) (P2-11.007)Neurology · 2024
13. Assessment of Barriers to Referral and Appointment Wait Times for the Evaluation of Spinal Muscular Atrophy (SMA): Findings from a Web-Based Physician SurveyNeurology and Therapy · 2024
14. Evaluating Perceived Fatigue within an Adult Spinal Muscular Atrophy PopulationNeurology and Therapy · 2023
15. A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence ScaleJournal of Neuromuscular Diseases · 2023
16. Telemedicine Use, Comfort, and Perceived Effectiveness in the Spinal Muscular Atrophy CommunityTelemedicine Journal and e-Health · 2023
17. Identifying Biomarkers of Spinal Muscular Atrophy for Further DevelopmentJournal of Neuromuscular Diseases · 2023
18. Effects of the COVID-19 Pandemic on SMA Screening and Care: Physician and Community InsightsNeurology and Therapy · 2023
19. The inter-media agenda-setting influence of social media activism on Egyptian news sites: a case study of violence against women (VAW)Insights into Language Culture and Communication · 2023
20. Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophyPrenatal Diagnosis · 2022