-- Rooted in patient voice and scientific rigor, this research reflects Chiesi's long-standing role in advancing science for the lysosomal storage disorders community -- PARMA, Italy, Aug. 28, 2026 (GLOBE NEWSWIRE) -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced presentations at the Society of Inborn Errors of Metabolism (SSIEM) 2026 Annual Symposium, held August 25-28, 2026, in Helsinki, Finland. The company is proud to support 13 scientific abstracts across Fabry disease and alpha-mannosidosis, including 6 Chiesi-led presentations and 7 independent studies supported by Chiesi through scientific research grants and educational projects. These abstracts reflect the scope of the Company's scientific engagement with the lysosomal storage disorders (LSDs) and rare disease communities. These abstracts highlight the breadth of research advancing the understanding of Fabry disease and alpha-mannosidosis. Chiesi-led presentations include long-term Phase 3 data from the F60/BRILLIANCE trial, patient-reported outcomes from a cross-sectional survey in untreated Fabry disease, and research exploring earlier diagnosis and genotype–phenotype relationships in alpha-mannosidosis. Independent studies supported by Chiesi examine real-world clinical experience, disease biology, biomarkers and newborn screening. Together, this research contributes to a growing body of...

iECURE (DTIL)/b) reported updated clinical data from the ongoing Phase 1/2 trial of gene-insertion therapy ECUR-506 in neonatal-onset ornithine transcarbamylase (OTC) deficiency, presented at the SSIEM 2026 symposium. According to iECURE, dosing is complete in all three cohorts (low, intermediate, high), with nine participants treated.As of July 28, 2026, ECUR-506 was generally well tolerated across dose levels, with no unexpected treatment-related safety events, thrombotic microangiopathy, or infusion reactions. In the low-dose cohort, annualized hyperammonemic events were reduced by 57% (p=0.02) and hyperammonemic crises by 74% (p=0.01) versus pre-treatment. Liver biopsies in two low-dose participants and serum PCSK9 reductions supported targeted hepatic editing. One participant achieved a complete clinical response, discontinuing ammonia scavengers and protein restriction, while the other two showed clinical improvements.

PTC Therapeutics presented compelling Sephience data at the 2026 SSIEM, showcasing significant benefits for PKU patients, including diet flexibility and metabolic control.

JIMD Podcasts · Episode

iECURE (NASDAQ: DTIL), a clinical-stage genome editing company, announced that data from its ongoing OTC-HOPE trial of ECUR-506 in infants with neonatal-onset ornithine transcarbamylase (OTC) deficiency will be featured in an oral presentation at the SSIEM Annual Symposium, held August 25-28, 2026, in Helsinki, Finland.The talk will present updated safety observations, genome editing data on metabolic control from the low-dose cohort, and analyses of baseline disease severity versus clinical response. The oral presentation, titled “Decreased rate of hyperammonemic crises in infants with neonatal-onset OTC deficiency post ECUR-506 administration (O-124),” is scheduled for Thursday, August 27, 3:30–5:00 p.m. EEST in Hall 5B, delivered by Julien Baruteau, M.D., Ph.D. iECURE will also exhibit at Booth B16, where its medical team will discuss the OTC-HOPE study and genome editing platform.