hyper rapid progression in salmonella associated mycotic aortic aneurysms a narrative review

Original title: “Hyper-rapid progression in Salmonella-associated mycotic aortic aneurysms: a narrative review.”

de winter wellens continuum evidence of dynamic bidirectional coronary collateral flow in acute coronary syndrome

Original title: “The de Winter-Wellens Continuum: Evidence of Dynamic Bidirectional Coronary Collateral Flow in Acute Coronary Syndrome.”

patient perspectives on late onset pompe disease insights from a 2025 patient snapshot survey on diagnosis treatment and quality of life

Original title: “Patient Perspectives on Late-Onset Pompe Disease: Insights From a 2025 Patient Snapshot Survey on Diagnosis, Treatment, and Quality of Life.”

past present and emerging antifungals against fusarium and other rare non aspergillus hyalohyphomycetes a narrative review

Original title: “Past, present, and emerging antifungals against Fusarium and other rare non-aspergillus hyalohyphomycetes: a narrative review.”

population based epidemiology of autoimmune seizures and epilepsies

Original title: “Population-Based Epidemiology of Autoimmune Seizures and Epilepsies.”

The Company reports that this work ... across therapeutic areas including immunology, oncology, infectious disease, paediatric and ophthalmology, highlighting potential applications in orphan diseases. The Company believes these findings are particularly significant given the increasing regulatory and commercial incentives associated with rare disease development. Programs targeting rare indications may benefit from expedited regulatory pathways, smaller and more efficient clinical trial designs, and ... The Company reports that this work has generated a number of highly differentiated and statistically significant matches across therapeutic areas including immunology, oncology, infectious disease, paediatric and ophthalmology, highlighting potential applications in orphan diseases. The Company believes these findings are particularly significant given the increasing regulatory and commercial incentives associated with rare disease development. Programs targeting rare indications may benefit from expedited regulatory pathways, smaller and more efficient clinical trial designs, and enhanced market exclusivity, which together can materially reduce development timelines and capital requirements. Screening against 1,700 rare disease signatures produced statistically significant matches. Next: small preclinical studies to build partner-ready data packages. Orphan diseases are rare medical conditions that affect a small number of patients compared with common illnesses. For investors, they matter because treatments for these conditions often receive special regulatory incentives—like tax credits, faster review, and market exclusivity—so a successful therapy can win a protected, premium-priced niche market much like a specialized product that serves few customers but commands high margins. The analysis evaluated CDT assets including its solid-form patent portfolio and licensed AstraZeneca compounds AZD1656 and AZD5658 against about 1,700 rare-disease signatures. CDT plans targeted preclinical validation to build out datasets for early-stage out-licensing discussions. Loading... Loading translation... Signature mapping produced multiple statistically significant matches across several therapeutic areas

Original title: “AI scan links CDT assets to rare diseases with few treatment options”

first generation proteolysis targeting chimeras protacs for the treatment of progeria

Original title: “First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria.”

wbc hbp dissociation unveils occult xanthogranulomatous inflammation mimicking hepatocellular carcinoma in diabetic patients

Original title: “"WBC-HBP Dissociation" Unveils Occult Xanthogranulomatous Inflammation Mimicking Hepatocellular Carcinoma in Diabetic Patients.”

two cases of emphysematous cystitis with urinary bladder rupture

Original title: “Two cases of emphysematous cystitis with urinary bladder rupture.”

tracking early cystic fibrosis in the new era

Original title: “Tracking early cystic fibrosis in the new era.”

artificial intelligence in hepatology a position paper by the italian association for the study of the liver

Original title: “Artificial Intelligence in hepatology: A position paper by the Italian Association for the Study of the Liver.”

menkes disease another example of progress in the development of pharmaceuticals for rare childhood diseases but much still remains to be done

Original title: “Menkes Disease: Another Example of Progress in the Development of Pharmaceuticals for Rare Childhood Diseases, But Much Still Remains to Be Done.”

pediatric covid 19 in korea lessons and strategies for future disease x preparedness

Original title: “Pediatric COVID-19 in Korea: Lessons and Strategies for Future Disease-X Preparedness.”

treatment discontinuation in patients with myelin oligodendrocyte glycoprotein antibody associated disease

Original title: “Treatment Discontinuation in Patients With Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease.”

real word evidence on healthcare resource use and associated costs in on demand users of replacement therapies in von willebrand disease in france the forvward study

Original title: “Real-word evidence on healthcare resource use and associated costs in on-demand users of replacement therapies in von Willebrand disease in France: the FORvWARD study.”

meta analysis ppar agonists for pruritus and quality of life in primary biliary cholangitis

Original title: “Meta-Analysis: PPAR Agonists for Pruritus and Quality of Life in Primary Biliary Cholangitis.”

heterozygous loss of function variant in mettl5 is associated with intellectual disability

Original title: “Heterozygous loss-of-function variant in METTL5 is associated with intellectual disability.”

open randomised withdrawal study of long term azithromycin in paediatric non cystic fibrosis bronchiectasis

Original title: “Open randomised withdrawal study of long-term azithromycin in paediatric non-cystic fibrosis bronchiectasis.”