Irish Health Service objectives state that patients with rare diseases should have timely access to genomic diagnostics with appropriate pre and post-test counselling. However, waiting times for clinical genetics outpatient appointments, during the study period, were up to two years as staffing levels remain low. A targeted public online survey was conducted in January 2022 to capture the experiences of Rare Disease families trying to access genetic testing and clinical genetic clinics in the Irish Republic. Irish patients experience significant waiting times to access clinical genetic services and self-report anxiety and stress, related to delayed access to diagnosis, clarity around recurrence risk and follow-up management. This negatively impacts personal decisions around family planning, education and employment and has a significant impact on family members seeking clarity on their own risk. Mainstream genetic testing activity is significant. Families report concern over the competency of health care professionals arranging and delivering genetic results and delays in accessing clinical genetics expertise to take them through the clinical implications. Timely access to clinical genetics expertise is important to ensure families with rare diseases have an appropriate understanding of the medical and reproductive implications of a genetic diagnosis and access to relevant care pathways. A national framework to develop competency in genomic literacy for health-care professionals including a national genetic test directory may be beneficial. Clinical genetics teams require ongoing support and investment to ensure the delivery of a safe and effective service for Irish families with rare diseases.
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http://dx.doi.org/10.1007/s12687-023-00664-w | DOI Listing |
Mol Inform
January 2025
Institute of Pharmaceutical and Medicinal Chemistry, University of Münster, Corrensstr. 48, 48149, Muenster, Germany.
Primary carnitine deficiency (PCD) is a rare autosomal recessive genetic disorder caused by missense mutations in the SLC22A5 gene encoding the organic carnitine transporter novel type 2 (OCTN2). This study investigates the structural consequences of PCD-causing mutations, focusing on the N32S variant. Using an alpha-fold model, molecular dynamics simulations reveal altered interactions and dynamics suggesting potential mechanistic changes in carnitine transport.
View Article and Find Full Text PDFHepatic ischemia-reperfusion injury (IRI) poses a significant threat to clinical outcomes and graft survival during hemorrhagic shock, hepatic resection, and liver transplantation. Current pharmacological interventions for hepatic IRI are inadequate. In this study, we identified ginsenoside Rk2 (Rk2), a rare dehydroprotopanaxadiol saponin, as a promising agent against hepatic IRI through high-throughput screening.
View Article and Find Full Text PDFEur J Radiol Open
June 2025
Department of Radiological Nuclear and Laboratory Medicine - Pisa University Hospital, Via Paradisa 2, Pisa 56124, Italy.
Since rare pancreatic cystic tumors may differ from common pancreatic cystic neoplasms in terms of treatment plan and prognosis, the differential diagnosis of these diseases is clinically relevant. Various imaging tests play an important role in the differential diagnosis of rare cystic pancreatic tumors, but accurately distinguishing these diseases solely on the basis of imaging findings is challenging. The purpose of this pictorial review is to present CT and in particular MR imaging features of rare pancreatic cystic tumors and discuss potential elements for differential diagnosis.
View Article and Find Full Text PDFHeliyon
January 2025
Department of Gastroenterology, Ganzhou People's Hospital, Ganzhou, Jiangxi, 341000, China.
Introduction: Bowel perforation due to migrated biliary stent is a rare complication. Here, we report a case of duodenal and ascending colonal perforation due to biliary stent migration.
Case Presentation: A 35-year-old man is complaining of right upper abdominal pain presented to the gastroenterology department.
Heliyon
January 2025
Department of Molecular Medicine, Sapienza University of Rome, Rome, Italy.
The 3p21.31 locus is the most robust genomic region associated with COVID-19 severity. This locus contains a main chemokine receptor (CKR) cluster.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!