GLP-1 receptor agonists as promising disease-modifying agents in WFS1 spectrum disorder

Front Clin Diabetes Healthc. 2023 Jun 2;4:1171091. doi: 10.3389/fcdhc.2023.1171091. eCollection 2023. ABSTRACT WFS1 spectrum disorder (WFS1-SD) is a rare monogenic neurodegenerative disorder whose cardinal symptoms are childhood-onset diabetes mellitus, optic atrophy, deafness, diabetes insipidus, and neurological signs ranging from mild to severe. The prognosis is poor as most patients die prematurely with severe neurological disabilities […]

Repetitive Suicidal Behaviors in a Case With a New Mutation of Wolfram Syndrome: A Jump From the Gene to the Behavior

Basic Clin Neurosci. 2022 Nov-Dec;13(6):893-900. doi: 10.32598/bcn.2021.910.3. Epub 2022 Nov 1. ABSTRACT Wolfram syndrome (WS) is a rare autosomal recessive neurodegenerative disease with variable symptoms, including neuropsychiatric manifestations. A 26-year-old man was reported with classic symptoms of WS and repetitive psychiatric hospitalizations and at least 16 suicidal attempts. The genetic study demonstrated a novel homozygous […]

Next Generation Sequencing (NGS) Target Approach for Undiagnosed Dysglycaemia

Life (Basel). 2023 Apr 24;13(5):1080. doi: 10.3390/life13051080. ABSTRACT Next-generation sequencing (NGS) has revolutionized the field of genomics and created new opportunities for basic research. We described the strategy for the NGS validation of the “dysglycaemia panel” composed by 44 genes related to glucose metabolism disorders (MODY, Wolfram syndrome) and familial renal glycosuria using Ion AmpliSeq […]

Clinical management and obstetric outcome in WFS1 Wolfram syndrome spectrum disorder: A case report and literature review

Taiwan J Obstet Gynecol. 2023 May;62(3):440-443. doi: 10.1016/j.tjog.2022.12.011. ABSTRACT OBJECTIVE: Wolfram Syndrome (WS) is a rare autosomal recessive neurodegenerative disorder caused by mutations in WFS1 or CISD2 (WFS2). We present a rare case report of pregnancy with WFS1 spectrum disorder (WFS1-SD) in our hospital and reviewed literature to provide the management of pregnancy in these […]

Wolfram syndrome: new pathophysiological insights and therapeutic strategies

Ther Adv Rare Dis. 2021 Aug 16;2:26330040211039518. doi: 10.1177/26330040211039518. eCollection 2021 Jan-Dec. ABSTRACT Wolfram Syndrome (WS) is an ultra-rare, progressive neurodegenerative disease characterized by early-onset diabetes mellitus and irreversible loss of vision, secondary to optic nerve degeneration. Visual loss in WS is an important cause of registrable blindness in children and young adults and the […]

Depletion of WFS1 compromises mitochondrial function in hiPSC-derived neuronal models of Wolfram syndrome

Stem Cell Reports. 2023 May 9;18(5):1090-1106. doi: 10.1016/j.stemcr.2023.04.002. ABSTRACT Mitochondrial dysfunction involving mitochondria-associated ER membrane (MAM) dysregulation is implicated in the pathogenesis of late-onset neurodegenerative diseases, but understanding is limited for rare early-onset conditions. Loss of the MAM-resident protein WFS1 causes Wolfram syndrome (WS), a rare early-onset neurodegenerative disease that has been linked to mitochondrial […]