30 closest matches · ranked by relevance
Myasthenic Syndromes, Congenital
Primary: Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis — -0.13; -1.75; -0.75; -1.14 Scores on a scale…
Congenital Myotonic Dystrophy
Primary: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) — -1.65; -3.40 score on a scale — p=0.0514
Myasthenia Gravis · Myasthenia Gravis, Juvenile Form · Myasthenia Gravis, Generalized
Primary: Change From Baseline in the QMG Total Score at Week 26 Regardless of Rescue Treatment — -6.1 units on a scale — p=0.0004
Neuromuscular Disease
Primary: Urine 15-F2t Isoprostane Concentration — 2.7; 2.6 ng/mg Cr — p=0.88
Myasthenia Gravis
Primary: Number of Patients With Treatment Emergent Adverse Events (TEAES) and Treatment Emergent Serious Adverse Events (SAEs) — 10; 10; 8; 3 Participants
Myasthenia Gravis
Primary: Time-weighted Average Quantitative Myasthenia Gravis Weakness Score Over 3 Years — 6.15; 8.99 units on a scale
Myasthenia Gravis
Primary: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Score to Visit 9 — -1.2; -1.8 scores on a scale — p==0.221
Myasthenia Gravis, Generalized
Primary: Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score. — -4.6; -2.7 units on a scale
Muscle Weakness
Primary: Percent Change in the Amplitude of Evoked Compound Muscle Action Potential by Electromyography Between the First to Fifth Response — 0 percent change
Myasthaenia Gravis
Primary: Mean Change From Baseline for Quantitative Myasthenia Gravis (QMG) Score at Week 24 — -2.37; -4.21 Units on a scale — p=0.256
Generalized Myasthenia Gravis
Primary: Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population — 67.7; 29.7 percentage of patients — p=<0.0001
Myasthenia Gravis, Generalized
Primary: Mean Change From Baseline in the Quantitative Myastenia Gravis (QMG) Score at Week 25. Posterior Median Was Used as Measure Type. — -4.07; -2.93 score
Myasthenia Gravis
Primary: Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 or Higher TEAEs, and Adverse Event (AE) Leading to…
Refractory Generalized Myasthenia Gravis
Primary: Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA)…
Myasthenia Gravis
Primary: To Determine the Effect of Acupuncture on Quality of Life in Patients With MG — 10.8; 9.4; 3.6; 8.3 score on a scale
Myasthenia Gravis
Primary: Percent of Subjects Achieving a 50% or Greater Reduction in CS Dose (Prednisone or Equivalent) From Baseline to Week 39 — 60.0; 63.3; 58.6; 60.7 percentage of subjects…
Generalized Myasthenia Gravis
Primary: Main Portion: Change From Baseline to Week 12 in Quantitative Myasthenia Gravis (QMG) Score — -5.5; -6.0; -3.2 score on a scale — p==0.0941
Myasthenia Gravis
Primary: Total Prednisone Dose Area Under the Curve — 2996.6; 3484.7 mg*Months — p=0.26
Myotonia · Non-Dystrophic Myotonia
Primary: Patient-reported Stiffness on the IVR — 2.53; 4.21; 1.60; 5.27 units on a scale — p=<0.001
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome · Chronic Fatigue Syndrome · Myalgic Encephalomyelitis
Primary: Change in Peak Oxygen Uptake (Peak VO2) Between the First and Second iCPET — 13.3; -40.3; 111.8 mL/min — p=0.043
Myasthenia Gravis Exacerbations
Primary: Change in Quantitative Myasthenia Gravis (QMG) Scale Score — -6.4 score on a scale — p=<0.001
DiGeorge Syndrome · Complete Typical DiGeorge Anomaly
Primary: Survival at 1 Year Post-Cultured Thymus Tissue Implantation (CTTI) — 72 % of participants who survive to 1 year
Spinal Muscular Atrophy (SMA)
Primary: Motor Function Assessments- Test for Infant Motor Performance Screening Items (TIMPSI) — 36.06; 88.47; 40.54; 89.44 scores on a scale
Generalized Myasthenia Gravis
Primary: Percentage of Patients With Quantitative MG Score (QMG) Score ≤ 4 and a Daily Prednisolon Dose of ≤ 10mg at 16 Weeks After Administration of Study Drug/Placebo. — 17; 6…
Acute Respiratory Failure
Primary: The Primary Outcome Variable for This Study Will be the Short Form of the Continuous Scale Physical Functional Performance Test (CS-PFP) Called the PFP-10 — 19.0; 20.9…
Generalized Myasthenia Gravis
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in…
Generalized Myasthenia Gravis
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) as a Measure of Safety and Tolerability — 11; 12; 9; 12 Participants
Myasthenia Gravis, Generalized
Primary: Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results — 4.96; 3.86; 6.14…
Congenital Muscular Dystrophy
Primary: Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil — 1.05; 4.75; 2.15; 3.27 ng/mL
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm