Evaluating the use of GLP-1 receptor agonists in Wolfram syndrome patients
Front Endocrinol (Lausanne). 2026 Jul 30;17:1847818. doi: 10.3389/fendo.2026.1847818. eCollection 2026. ABSTRACT INTRODUCTION: Wolfram syndrome is a rare autosomal recessive disorder caused by pathogenic variants in the WFS1 gene and characterized by early-onset, insulin-dependent diabetes mellitus, optic atrophy, sensorineural hearing loss, arginine vasopressin deficiency, and progressive neurodegeneration driven by chronic endoplasmic reticulum (ER) stress; no proven […]
Exploratory Associations Between Multimodal MRI-Derived Features and Neurological Symptoms in Wolfram Syndrome: A Spanish Cohort Pilot Study
Diagnostics (Basel). 2026 Jul 30;16(15):2396. doi: 10.3390/diagnostics16152396. ABSTRACT Background/Objectives: Wolfram syndrome is an ultra-rare, progressive multisystem disorder in which endocrine and sensory manifestations coexist with neurological involvement. Quantitative magnetic resonance imaging (MRI) may help characterize central nervous system involvement in this condition; however, evidence derived from small imaging cohorts requires cautious interpretation. This study aimed […]
Global trends and hotspots in diabetes and hearing loss: a 20-year systematic bibliometric study
Front Med (Lausanne). 2026 Jul 28;13:1867924. doi: 10.3389/fmed.2026.1867924. eCollection 2026. ABSTRACT BACKGROUND: Diabetes and hearing loss are increasingly recognized as critical global public health concerns. Cumulative evidence indicates a close association between diabetes and auditory impairment; however, the knowledge structure, research hotspots, and developmental trends in this field remain insufficiently and systematically characterized. This systematic […]
Genotype-based severity scoring system in Wolfram Syndrome: correlation with onset of cardinal symptoms and WFS1 gene variant types
Front Genet. 2026 Jul 15;17:1839135. doi: 10.3389/fgene.2026.1839135. eCollection 2026. ABSTRACT BACKGROUND: Wolfram syndrome is a rare genetic disorder characterized by antibody-negative early-onset atypical diabetes mellitus, optic nerve atrophy, sensorineural hearing loss, central diabetes insipidus (arginine vasopressin deficiency), and progressive neurodegeneration, with significant variability in disease severity. We assessed the accuracy of a genotype-based severity scoring […]
Correction to “Wolfram Syndrome-2, a cause of severe gastrointestinal bleeding: A case series and a literature review”
JPGN Rep. 2026 May 7. doi: 10.1002/jpr3.70193. Online ahead of print. ABSTRACT [This corrects the article DOI: 10.1097/PG9.0000000000000339.]. PMID:42499749 | PMC:PMC13398631 | DOI:10.1002/jpr3.70193
Consensus Recommendations for the Clinical Management of Wolfram syndrome Using a Delphi Method
medRxiv [Preprint]. 2026 Jul 2:2026.07.02.26357130. doi: 10.64898/2026.07.02.26357130. ABSTRACT BACKGROUND: Wolfram syndrome is a rare neurodegenerative disorder, most commonly caused by pathogenic variants in WFS1 , while cases due to CISD2 are exceedingly rare. The estimated prevalence is 1 in 160,000 to 770,000 individuals worldwide. In these clinical guidelines, disorders caused by WFS1 are referred to […]
Revision of diagnosis in a child with type 1 diabetes mellitus due to appropriate genetic testing – Wolfram Syndrome
BMJ Case Rep. 2026 Jul 2;19(7):e267549. doi: 10.1136/bcr-2025-267549. ABSTRACT We present a middle childhood boy with type 1b diabetes mellitus (defined as insulin-dependent diabetes mellitus without evidence of autoimmunity) diagnosed at the age of 4 years who was on a basal bolus insulin regimen. Identifying the presence of bilateral optic atrophy in him during an […]
Reversible Mitochondrial Iron Toxicity in Wolfram Syndrome Type 2 Monogenic Diabetes
J Clin Endocrinol Metab. 2026 Jun 24:dgag250. doi: 10.1210/clinem/dgag250. Online ahead of print. ABSTRACT CONTEXT: Wolfram syndrome type 2 (WS2) is a rare monogenic diabetes syndrome caused by CISD2 mutations. Its cellular pathophysiology remains poorly understood, and no targeted therapies exist. OBJECTIVE: To characterize the clinical phenotype and cellular pathophysiology of the largest WS2 cohort […]
Wolfram syndrome and diabetes mellitus in Aotearoa, New Zealand: Phenotype and response to GLP-1 receptor agonist therapy
Diabet Med. 2026 Jun 21:e70405. doi: 10.1111/dme.70405. Online ahead of print. ABSTRACT AIM: To describe the clinical characteristics of patients with Wolfram syndrome (WFS) and diabetes mellitus (DM) in Aotearoa, New Zealand. Review of response to therapy in those treated with glucagon-like peptide-1 receptor agonists (GLP1RA). METHODS: This retrospective cohort study describes 7 patients with […]
Targeting WFS1 overcomes KRASG12D dependency and adaptive resistance to KRAS inhibition in pancreatic cancer
NPJ Precis Oncol. 2026 Jun 15. doi: 10.1038/s41698-026-01553-4. Online ahead of print. ABSTRACT KRASG12D-driven pancreatic ductal adenocarcinoma (PDAC) remains a therapeutic challenge characterized by limited treatment options. While MRTX1133, a potent and selective KRASG12D inhibitor, is currently under clinical evaluation, the emergence of acquired resistance restricts its long-term therapeutic efficacy. In this study, we identified […]