123 results on '"Day, John W."'
Search Results
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
4. Adeno-Associated Virus Serotype 9 Antibody Seroprevalence for Patients in the United States with Spinal Muscular Atrophy
5. Generation of two induced pluripotent stem cell lines from Duchenne muscular dystrophy patients
6. Generation of two induced pluripotent stem cell lines from spinal muscular atrophy type 1 patients carrying no functional copies of SMN1 gene
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
12. Coastal wetland area change for two freshwater diversions in the Mississippi River Delta
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
15. A review of issues related to formation, deterioration and restoration of the Chenier Plain, Mississippi River Delta, LA - Combining nature based and engineered approaches
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
17. A review of technologies for closing the P loop in agriculture runoff: Contributing to the transition towards a circular economy
18. The avalglucosidase alfa phase 3 COMET trial in late-onset Pompe disease patients: Efficacy and safety results after 97 weeks
19. AT845 gene replacement therapy for late onset Pompe disease: Overview of clinical data from FORTIS, a phase 1/2 open-label clinical study
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
23. Advances in the Therapy of Spinal Muscular Atrophy
24. Treatment of infantile-onset spinal muscular atrophy with nusinersen: final report of a phase 2, open-label, multicentre, dose-escalation study
25. Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec
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
28. A phase I/II open-label gene replacement clinical study for late onset Pompe disease
29. Transcriptome alterations in myotonic dystrophy frontal cortex
30. Elevation and accretion dynamics at historical plots in the Biloxi Marshes, Mississippi Delta
31. A review of emerging organic contaminants (EOCs), antibiotic resistant bacteria (ARB), and antibiotic resistance genes (ARGs) in the environment: Increasing removal with wetlands and reducing environmental impacts
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
35. Above- and belowground response of baldcypress and water tupelo seedlings to variable rates of nitrogen loading: Mesocosm and field studies
36. Canals, backfilling and wetland loss in the Mississippi Delta
37. Municipal wastewater treatment costs with an emphasis on assimilation wetlands in the Louisiana coastal zone
38. Long-term assimilation wetlands in coastal Louisiana: Review of monitoring data and management
39. A review of sediment diversion in the Mississippi River Deltaic Plain
40. Consequences of Mississippi River diversions on nutrient dynamics of coastal wetland soils and estuarine sediments: A review
41. Drivers and impacts of water level fluctuations in the Mississippi River delta: Implications for delta restoration
42. Can denitrification explain coastal wetland loss: A review of case studies in the Mississippi Delta and New England
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
44. Evaluating trade-offs of a large, infrequent sediment diversion for restoration of a forested wetland in the Mississippi delta
45. Modeling impacts of sea-level rise, oil price, and management strategy on the costs of sustaining Mississippi delta marshes with hydraulic dredging
46. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study
47. The impact of two large floods (1993–1994) on sediment deposition in the Rhône delta: Implications for sustainable management
48. Large infrequently operated river diversions for Mississippi delta restoration
49. Approaches to defining deltaic sustainability in the 21st century
50. Enhancing mud supply from the Lower Missouri River to the Mississippi River Delta USA: Dam bypassing and coastal restoration
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