INZY - Inozyme Pharma reports data from burden of illness study in fatal genetic diseases

Inozyme Pharma (INZY) presents data that highlight the burden of disease for patients and families affected by ENPP1 and ABCC6 Deficiency, two potentially deadly genetic diseases.A total of 38 respondents participated in the study. The most frequently reported burdens for patients with ENPP1 Deficiency at all time points were:Bone and joint pain (100% of adults, 85% of pediatric patients)Cardiac issues (86% of adults, 85% of pediatric patients)Mobility issues/fatigue (86% of adults, 85% of pediatric patients) The most frequently reported symptoms for patients with ABCC6 Deficiency were gastrointestinal issues (83%), growth and development issues (83%) and cardiac issues (67%). ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy ((GACI)) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2).ABCC6 Deficiency is a rare, inherited disorder caused by mutations in the ABCC6 gene, resulting in decreased or absent activity of the ABCC6 protein, resulting

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Inozyme Pharma reports data from burden of illness study in fatal genetic diseases