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Corporate Presentation

Rare diseases CHI Congenital Hyperinsulinism (CHI) is a severe, ultra-rare genetic disorder with significant impact on patients' QoL ZEAL& ZEALAND PHARMA CHI is an ultra-rare disease in newborns and children • • 1 in 28-50,000 newborns per year are diagnosed with genetically determined CHI in the US and EU1,2 CHI can cause serious episodes of hypoglycemia during childhood 2,3 Persistent episodes of hypoglycemia may result in brain damage • Hypoglycemia can cause seizures in ~50% of the patients4 Lack of proper management within days can lead to permanent brain injury and neurocognitive impairment 3.4 Significant impact on the patient and caregivers' quality of life . • Complex care requirements can cause lengthy and frequent hospitalizations and make daily social activities difficult 4,5 Severe CHI requires continuous enteral feeding, making transfer to other caregivers difficult (e.g., school)4 More than 50% of CHI patients may be unresponsive to current medical treatment options6 1 Arnoux JB et al. 2011 Orphanet J Rare Dis;6:63; 2 Yau et al. Plos One 2020;15(2):e0228417; ³Thornton PS et al., J Pediatr. 2015;167(2):238-45. 4Banerjee I et al., Orphanet J Rare Dis. 2022;17:61; 5Pasquini TLS et al. Front Endocrinol 2022:13;876903; 6Yorifuji et al. Clin Pediatr Endocrinol 2017;26(3):127-152. 34
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