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Gaucher disease in children
Gaucher disease is an autosomal recessive lysosomal storage disorder caused by pathogenic variants in the GBA1 gene, resulting in deficient activity of the enzyme acid β glucosidase.

Efficacy and safety of dupilumab in patients with PN with or without a history of atopic comorbidities

DUPIREAL: Two-Year Turning Point with Dupilumab in CRSwNP
The results from the DUPIREAL real-world study reinforce the efficacy of dupilumab demonstrated in the SINUS-24 and SINUS-52 randomized clinical trials and are consistent with the approved SmPC.1,2,3,4 DUPIREAL is the largest long-term real-world study (n=926) evaluating the effectiveness of dupilumab, including treatment response, disease control and remission, in severe uncontrolled CRSwNP over 2 years.2,3

Real-World Evidence and Phase IV studies in CRSwNP
Patients with chronic rhinosinusitis with nasal polyps (CRSwNP) generally have a high symptom burden and poor health-related quality of life (QoL), often requiring recurring systemic corticosteroid use and repeated sinus surgery.

Atopisch Eczeem bij kinderen

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Mechanism of Disease
Complex immune dysregulation can lead to autoimmunity and inflammation1-6

Living with ITP
Patients reported significant quality of life (QoL) challenges related to their ITP1