69 results on '"Day, John"'
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2. Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study
3. A longitudinal study of CMT1A using Rasch analysis based CMT neuropathy and examination scores
4. Assessment of disease progression in dysferlinopathy: A 1-year cohort study
5. Mesial Temporal Enlargement in Adult-Onset Myotonic Dystrophy Type 1 (P1-1.Virtual)
6. Study Design of AOC 1001-CS1, a Phase 1/2 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Patients with Myotonic Dystrophy Type 1 (DM1) (MARINA) (S23.006)
7. Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005)
8. Quantitative muscle strength changes in adults with Spinal Muscular Atrophy receiving Nusinersen (P5-13.008)
9. The RESTORE Registry: Real-World Assessments of Interventions and Long-Term Outcomes in Patients with Spinal Muscular Atrophy (SMA) (P15-5.002)
10. Prevalence of healthcare conditions and services used by patients with myotonic dystrophy (DM1 and DM2) prior to diagnosis; a real-world data analysis (P14-13.003)
11. Pooled Safety Data from the Risdiplam Clinical Trial Development Program (P18-5.001)
12. FIREFISH Parts 1 and 2: 24-month Safety and Efficacy of Risdiplam in Type 1 SMA (S39.005)
13. Exploring Protein Changes in Cerebrospinal Fluid of Spinal Muscular Atrophy Patients Pre-Nusinersen vs. Post-Nusinersen Treatment using Bayesian Machine Learning Algorithms (P1-1.Virtual)
14. SUNFISH Part 2: 24-month Efficacy and Safety of Risdiplam in Patients with Type 2 or Non-ambulant Type 3 Spinal Muscular Atrophy (SMA) (2240)
15. Efficacy and Safety Results of the Avalglucosidase alfa Phase 3 COMET Trial in Late-Onset Pompe Disease Patients (4195)
16. Results of Double-blind, Placebo-controlled, Dose Range Finding, Crossover Study of Single Day Administration of ERX-963 (IV Flumazenil) in Adults with Myotonic Dystrophy Type 1 (2834)
17. A phase I/II open-label gene replacement clinical study for late onset Pompe disease (4399)
18. Pooled Safety Data from the Risdiplam Clinical Trial Development Program (4184)
19. Escalating Dose and Randomized, Controlled Study of Nusinersen in Participants With Spinal Muscular Atrophy (SMA); Study Design and Part A Data for the Phase 2/3 DEVOTE (232SM203) Study to Explore High Dose Nusinersen (2343)
20. Long-Term Follow-Up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA) (2301)
21. Onasemnogene Abeparvovec-xioi Gene-Replacement Therapy for Spinal Muscular Atrophy Type 1 (SMA1): Phase 3 US Study (STR1VE) Update (1828)
22. Real-World Treatment Patterns and Outcomes in Patients with Spinal Muscular Atrophy Collected from the RESTORE Registry (2470)
23. JEWELFISH: Safety and Pharmacodynamic Data in Non-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (RG7916) (772)
24. Longer-term Experience with Nusinersen in Teenagers and Young Adults with Spinal Muscular Atrophy: Results from the CS2/CS12 and SHINE Studies (1132)
25. Escalating Dose and Randomized, Controlled Study of Nusinersen in Participants with Spinal Muscular Atrophy; Study Design of the Phase 2/3 DEVOTE (232SM203) Study to Explore High Dose Nusinersen (1169)
26. SUNFISH Part 2: Efficacy and Safety of Risdiplam (RG7916) in Patients with Type 2 or Non-Ambulant Type 3 Spinal Muscular Atrophy (SMA) (1260)
27. Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy Through Newborn Screening (5214)
28. One-Time Intrathecal (IT) Administration of AVXS-101 IT Gene-Replacement Therapy for Spinal Muscular Atrophy: Phase 1 Study (STRONG) (2493)
29. FIREFISH Part 1: Survival, Ventilation and Swallowing Ability in Infants with Type 1 SMA Receiving Risdiplam (RG7916) (S25.008)
30. FIREFISH Part 1: 1-Year Results on Motor Function in Babies with Type 1 SMA (S25.003)
31. AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 1 (SMA1): Pivotal Phase 3 Study (STR1VE) Update (P1.6-058)
32. Nusinersen Efficacy in Adults with Spinal Muscular Atrophy (S58.007)
33. Phase 1 Study of Intrathecal Administration of AVXS-101 Gene-Replacement Therapy (GRT) for Spinal Muscular Atrophy Type 2 (SMA2) (STRONG) (P1.6-059)
34. Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) Feasibility for Individuals with Severe Spinal Muscular Atrophy II (S46.004)
35. Disease progression in Myotonic Dystrophy Type 1 during a non-interventional multicenter study (S22.004)
36. Nusinersen Efficacy in Adults with Spinal Muscular Atrophy (S46.001)
37. Examining longitudinal functional changes in Dysferlinopathy: The JAIN Clinical Outcome Study (P5.429)
38. Targeted Next Generation Sequencing (NGS) for Analysis of Splicing Biomarkers of Myotonic Dystrophy Type 1 (DM1) (S22.002)
39. Self-Reported Sleep Features in Myotonic Dystrophies (P5.438)
40. Increased EEG theta spectral power in polysomnography of myotonic dystrophy type 1 compared to matched controls. (P5.107)
41. Interim Results of a Phase 2 Clinical Study of Nusinersen (ISIS-SMNRx) in Patients with Infantile-Onset Spinal Muscular Atrophy (P5.004)
42. Should Motor Function Determine the Timing of Scoliosis Surgery in Spinal Muscular Atrophy (SMA)? (P5.006)
43. Ataluren: An Overview of Clinical Trial Results in Nonsense Mutation Duchenne Muscular Dystrophy (P3.162)
44. Study Design of a Phase 1/2a Trial with ISIS-DMPKRx for the Treatment of Myotonic Dystrophy Type 1 (P3.167)
45. Cross-sectional analysis of a large cohort with X-linked Charcot-Marie-Tooth disease (CMTX1).
46. Results of a Phase 2 Open-Label Study of ISIS-SMNRx in Patients with Infantile (Type 1) Spinal Muscular Atrophy (S6.003)
47. A Novel Form of Nemaline Myopathy Caused by an In-Frame Deletion in TNNT1 (P5.095)
48. Kenneth Ricker, MD (1935–2004)
49. A focal domain of extreme demethylation within D4Z4 in FSHD2.
50. Rapid resolution of quadriplegic CIDP by combined plasmapheresis and IVIg.
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