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123 results on '"Day, John W."'

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2. Sunfish parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)

3. The concept of land bridge marshes in the Mississippi River Delta and implications for coastal restoration

7. Expanding the muscle imaging spectrum in dysferlinopathy: description of an outlier population from the classical MRI pattern

8. Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial

9. Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy

10. Efficacy and safety of avalglucosidase alfa in participants with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

11. AT845 gene replacement therapy for late onset Pompe disease: An update on safety and preliminary efficacy data from FORTIS, a phase I/II open-label clinical study

13. O19: Integrated analyses of data from clinical trials of delandistrogene moxeparvovec gene therapy in DMD*

14. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial

16. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

18. The avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients: Efficacy and safety results after 97 weeks

20. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

21. Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients With Duchenne Muscular Dystrophy

22. Increased tissue stiffness triggers contractile dysfunction and telomere shortening in dystrophic cardiomyocytes

24. Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study

26. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial

27. Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease

29. Transcriptome alterations in myotonic dystrophy frontal cortex

32. Transcriptome Alterations in Myotonic Dystrophy Frontal Cortex

33. Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease

34. Wetland shear strength with emphasis on the impact of nutrients, sediments, and sea level rise

43. Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

46. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

49. Approaches to defining deltaic sustainability in the 21st century

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