Publications by authors named "Marion Afonso"
Introduction: Pompe disease is caused by a rare biallelic mutation in the GAA gene resulting in acid α-glucosidase deficiency and glycogen accumulation.
Aim: We analyzed hospital admissions associated with the administration of Myozyme®, utilizing the French hospital discharge database, known in France as the Programme de Médicalisation des Systèmes d'Information (PMSI), which comprehensively captures all hospital activity within the country.
Methods: In this observational study, we examined hospitalization records from April 4, 2012, to December 31, 2019, within the PMSI database, focusing on admissions where Myozyme® was administered.
Introduction: An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles.
Aim: We sought to understand how people with haemophilia (PwH) and their caregivers value the potential benefits of novel prophylactic treatments. We conducted a discrete-choice experiment (DCE) to quantify preferences for features of haemophilia treatments among adults and caregivers of children with haemophilia.
Article Synopsis
- Diabetes-related distress is prevalent among individuals with diabetes and negatively impacts both glycemic control and overall health, making it an important patient-reported outcome (PRO).
- A study analyzing data from adults with type 1 diabetes (T1D) using sotagliflozin alongside insulin found that a higher percentage of patients reported significant improvements in treatment satisfaction compared to those on placebo.
- Overall, patients taking sotagliflozin experienced notable reductions in both diabetes distress and improvements in treatment satisfaction, highlighting its potential benefits in diabetes management.
Objectives: This study investigated the cost per responder and number needed to treat (NNT) in type 2 diabetes mellitus (T2DM) patients for lixisenatide compared to insulin intensification regimens using composite endpoints in the UK, Italy, and Spain.
Methods: Efficacy and safety outcomes were obtained from GetGoal Duo-2, a 26-week phase 3 trial comparing lixisenatide vs insulin glulisine (IG) once daily (QD) and three times daily (TID). Response at week 26 was extrapolated to 52 weeks, assuming a maintained treatment effect, based on long-term evidence in other T2DM populations.