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1,419 results on '"Day, John"'

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2. Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA

5. Global dataset of soil organic carbon in tidal marshes

7. Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA)

8. Long-term efficacy and safety of a treatment strategy for HIV infection using protease inhibitor monotherapy: 8-year routine clinical care follow-up from a randomised, controlled, open-label pragmatic trial (PIVOT)

11. 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

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

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

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

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

21. Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes

23. Long-Range Imaging of Alpha Emitters Using Radioluminescence in Open Environments: Daytime and Night-Time Applications.

24. Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.

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

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

30. Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen

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

32. 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

33. Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

34. Advancements in Remote Alpha Radiation Detection: Alpha-Induced Radio-Luminescence Imaging with Enhanced Ambient Light Suppression.

35. Co-located ecological data for exploring top- and subsoil carbon dynamics across grassland-woodland contrasts.

36. Conformational fingerprinting with Raman spectroscopy reveals protein structure as a translational biomarker of muscle pathology.

37. Beyond Contractures in Spinal Muscular Atrophy: Identifying Lower-Limb Joint Hypermobility.

41. Venous Vascular Closure System Versus Manual Compression Following Multiple Access Electrophysiology Procedures: The AMBULATE Trial

48. Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry.

49. The intensification of parenting and generational fracturing of spontaneous physical activity from childhood play in the United Kingdom.

50. Telomere shortening is a hallmark of genetic cardiomyopathies

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