Publications by authors named "N Bedreddine"
Article Synopsis
- Fabry disease presents complex challenges in patient care, and while enzyme replacement therapy (agalsidase beta) is beneficial, its long infusion time can be burdensome for patients.
- A study involved 39 adult patients with Fabry disease to evaluate the safety of reducing the infusion time to 90 minutes without premedication, with a total of 85 infusions conducted.
- The findings showed no reported adverse events, stable vital signs, and high patient satisfaction, indicating that shorter infusion times are safe and feasible for patients with Fabry disease.*
Fabry disease is the second most frequent lysosomal storage disorder. It is a X-linked genetic disease secondary to alpha-galactosidase A enzyme deficiency. This is a progressive and systemic disease that affects both males and females.
View Article and Find Full Text PDFFabry disease is a frequent lysosomal storage disorder secondary to the deficiency of alpha-galactosidase A enzyme. This X-linked genetic disease realizes progressive and systemic manifestations that affect both male and female. Fabry disease may present as "classical", as "late-onset" or "non-classical" forms.
View Article and Find Full Text PDFBackground: Fabry disease (FD) is a rare, X-linked, inherited lysosomal disease caused by absent or reduced α-galactosidase A activity. Due to the heterogeneity of disease presentation and progression, generic patient-reported outcome (PRO) tools do not provide accurate insight into patients' daily lives and impact of disease specific treatments. Also, the French National Health Authority, (HAS) actively encourages a patient-centric approach to improve the quality of care throughout the patient journey.
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