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Population Pharmacokinetics of Trofinetide in a Pediatric Population Aged 2–4 Years with Rett Syndrome
Population Pharmacokinetics of Trofinetide in a Pediatric Population Aged 2–4 Years with Rett Syndrome
Weight-banded trofinetide dosing improved physician- and caregiver-rated efficacy measures and had acceptable tolerability...
The Urgency Numeric Rating Scale: Psychometric Evaluation in Adults with Crohn’s Disease
The Urgency Numeric Rating Scale: Psychometric Evaluation in Adults with Crohn’s Disease
Bowel urgency has recently been recognized as a Crohn’s disease (CD) symptom that substantially impacts patientsR...
Population Pharmacokinetic Modeling to Support Trofinetide Dosing for the Treatment of Rett Syndrome
Population Pharmacokinetic Modeling to Support Trofinetide Dosing for the Treatment of Rett Syndrome
Oral trofinetide administered using a body weight-banded dosing regimen was approved in the US for the treatment of Rett s...
An Indirect Treatment Comparison of Lenvatinib for the First-Line Treatment of Patients with Unresectable Hepatocellular Carcinoma
An Indirect Treatment Comparison of Lenvatinib for the First-Line Treatment of Patients with Unresectable Hepatocellular Carcinoma
This study compared the relative efficacy of first-line lenvatinib, a standard-of-care treatment for unresectable hepatoce...
Qualitative Assessment of Health-Related Quality of Life Impacts Associated with Sickle Cell Disease in the United States and United Kingdom
Qualitative Assessment of Health-Related Quality of Life Impacts Associated with Sickle Cell Disease in the United States and United Kingdom
Individuals living with sickle cell disease (SCD) commonly report impaired health-related quality of life (HRQoL). However...
Baseline Characteristics of Dupilumab-Treated Patients with Asthma in the Real World: The RAPID Global Registry
Baseline Characteristics of Dupilumab-Treated Patients with Asthma in the Real World: The RAPID Global Registry
Patients with uncontrolled, moderate-to-severe asthma have a higher risk for exacerbations, negatively impacting lung func...
One-Year Insights into the GLOBOSTAD Multinational Prospective Observational Study of Patients Receiving Dupilumab for Atopic Dermatitis
One-Year Insights into the GLOBOSTAD Multinational Prospective Observational Study of Patients Receiving Dupilumab for Atopic Dermatitis
Currently, limited data are available on long-term use of dupilumab to treat atopic dermatitis (AD) in a multinational rea...
Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice
Effects of Current Therapies on Disease Progression in Fabry Disease: A Narrative Review for Better Patient Management in Clinical Practice
Fabry disease (FD) is a rare lysosomal storage disorder that is characterized by renal, neurological, and cardiovascular d...
Genetic Etiologies and Outcomes in Malignancy and Mortality in Activated Phosphoinositide 3-Kinase Delta Syndrome: A Systematic Review
Genetic Etiologies and Outcomes in Malignancy and Mortality in Activated Phosphoinositide 3-Kinase Delta Syndrome: A Systematic Review
This analysis evaluated literature on patients with activated phosphoinositide 3-kinase delta syndrome (APDS) to better un...
A Pharmacoeconomic Study of Post-Exposure Prophylaxis Strategies for Influenza Virus Infections in Japan
A Pharmacoeconomic Study of Post-Exposure Prophylaxis Strategies for Influenza Virus Infections in Japan
Vaccines can prevent influenza (flu) infections and are cost-effective for society and healthcare. However, the cost-effec...
Influence of Treatment Effect Modifiers in Fabry Disease: A Systematic Literature Review
Influence of Treatment Effect Modifiers in Fabry Disease: A Systematic Literature Review
Fabry disease (FD) is a rare metabolic disorder which presents with considerable heterogeneity in disease characteristics....
Machine Learning Applications in Acute Coronary Syndrome: Diagnosis, Outcomes and Management
Machine Learning Applications in Acute Coronary Syndrome: Diagnosis, Outcomes and Management
Acute coronary syndrome (ACS) is a leading cause of death worldwide. Prompt and accurate diagnosis of acute myocardial inf...
Addressing the Human Experience of Chronic Kidney Disease: A Call to Transform Kidney Care
Addressing the Human Experience of Chronic Kidney Disease: A Call to Transform Kidney Care
Chronic kidney disease (CKD), a long-term condition in which kidney function declines over time, is a growing global healt...
Survival Improvement of Stage IV Non-small Cell Lung Cancer in the Immunotherapy Era: A Retrospective Cohort Study in a US Population
Survival Improvement of Stage IV Non-small Cell Lung Cancer in the Immunotherapy Era: A Retrospective Cohort Study in a US Population
Immune checkpoint inhibitors (ICIs) greatly improved outcomes of stage IV non-small cell lung cancer (NSCLC) in r...
Pathophysiology of Bullous Pemphigoid: Role of Type 2 Inflammation and Emerging Treatment Strategies (Narrative Review)
Pathophysiology of Bullous Pemphigoid: Role of Type 2 Inflammation and Emerging Treatment Strategies (Narrative Review)
Bullous pemphigoid (BP) is an autoimmune blistering disease that most often affects elderly individuals and has a signific...
Cost-Effectiveness of Teduglutide for Pediatric Patients with Short Bowel Syndrome in Japan, Including Caregiver Burden
Cost-Effectiveness of Teduglutide for Pediatric Patients with Short Bowel Syndrome in Japan, Including Caregiver Burden
Short bowel syndrome (SBS) is associated with a significant mental and physical burden for patients and caregivers. Standa...
Fluticasone Furoate/Umeclidinium/Vilanterol Initiation Following a COPD Exacerbation: Benefits of Prompt Initiation on COPD Outcomes
Fluticasone Furoate/Umeclidinium/Vilanterol Initiation Following a COPD Exacerbation: Benefits of Prompt Initiation on COPD Outcomes
Previous real-world evidence suggests that prompt versus delayed initiation of single-inhaler triple therapy (SITT) with f...