Publications by authors named "C Maggiore"
Introduction: Haemophilia A is an X-linked bleeding disorder resulting from a deficiency of factor VIII (FVIII). To date, multiple gene therapies have entered clinical trials with the goal of providing durable haemostatic protection from a single dose. TAK 754 (BAX 888) is an investigational AAV8-based gene therapy containing a FVIII transgene.
View Article and Find Full Text PDFPurpose: Neoadjuvant chemotherapy (NAC) has a well-established role in locally advanced or chemoresponsive breast cancers (BC). Chemotherapic regimens are effective when patients receive the optimal doses. Toxicities are common in overweight/obese patients but may occur also in normal weight counterparts.
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- Italy implemented mandatory newborn screening for spinal muscular atrophy (SMA) in late 2021, enabling early detection and treatment of infants.
- Analysis of DNA from newborn blood samples identified a small number of SMA cases, with infants diagnosed and treated as early as 7 days old.
- The early treatment approach was effective, as most infants remained asymptomatic and reached normal motor development milestones over 16 months of follow-up.
Background: Pain is a prevalent symptom among cancer patients, and its management is crucial for improving their quality of life. However, pain management in cancer patients referred to radiotherapy (RT) departments is often inadequate, and limited research has been conducted on this specific population. This study aimed to assess the adequacy and effectiveness of pain management when patients are referred for RT.
View Article and Find Full Text PDFImmunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study.
Expert Rev Hematol
November 2023
Aim: To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients (PUPs) with severe hemophilia A (HA).
Methods: This prospective, phase 3 study (NCT02615691) was conducted in PUPs, or patients with ≤2 exposure days (EDs) prior to screening, aged <6 years with severe HA. The primary endpoint was incidence of factor VIII (FVIII) inhibitor development.