
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.
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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.


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

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.



Complex immune dysregulation can lead to autoimmunity and inflammation1-6

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

Low platelet counts are one facet of ITP, but when we look at it from another angle, we see the multiple dimensions of ITP. ITP is a condition of complex immune dysregulation that reduces platelet counts—causing bleeding, bruising, fatigue, and other health-related quality-of-life (HRQoL) issues—while increasing inflammation and the risk of thromboembolic events (TEs).1-5