Patient-Reported Symptom Burden of Charcot-Marie-Tooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study.

Objectives: This study aims to explore the impact of Charcot-Marie-Tooth disease type 1A (CMT1A) and its treatment on patients in European (France, Germany, Italy, Spain, and the United Kingdom) and US real-world practice.

Methods: Adults with CMT1A (n = 937) were recruited to an ongoing observational study exploring the impact of CMT. Data were collected via CMT&Me, an app through which participants completed patient-reported outcome measures.

Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study.

Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and sensory neuropathy affecting the peripheral nervous system. This study will explore the real-world impact of CMT. The trial is a digital study of approximately 2000 people in 6 countries with CMT ≥18 years.

Effect of early initiation of statins on survival in patients with acute myocardial infarction (the USIC 2000 Registry).

We evaluated the association of statin initiation within 48 hours of admission for an acute myocardial infarction with a 1-year prognosis on a nationwide scale. Patients who received a statin within 48 hours of admission but not before hospitalization had an improved prognosis (hazard ratio 0.57, 95% confidence interval 0.

Impact of prehospital thrombolysis for acute myocardial infarction on 1-year outcome: results from the French Nationwide USIC 2000 Registry.

Background: Limited data are available on the impact of prehospital thrombolysis (PHT) in the "real-world" setting.

Methods And Results: Of 443 intensive care units in France, 369 (83%) prospectively collected all cases of infarction (< or =48 hours of symptom onset) in November 2000; 1922 patients (median age, 67 years; 73% men) with ST-segment-elevation infarction were included, of whom 180 (9%) received intravenous thrombolysis before hospital admission (PHT). Patients with PHT were younger than those with in-hospital thrombolysis, primary percutaneous interventions, or no reperfusion therapy.