8th International Wolfram Symposium Presentation Mathias Leinders – Amylyx Pharmaceuticals Helios – A Phase II Study of Safety and Efficacy of AMX0035 in Wolfram Syndrome Summary: Update on the clinical trial design of Helios, and the rationale for using AMX0035 as potential treatment for Wolfram Syndrome. Points noted: AMX0035 is a combination therapy of two […]
8th International Wolfram Symposium Presentation Dr Benjamin Delprat – University of Montpellier, France Advances on the pharmacological and gene therapies approaches Abstract: Wolfram syndrome is a rare autosomal recessive disease affecting many organs with life-threatening consequences and currently no treatment is available. Therefore, the need to find a cure is imperative. The pathology is […]
8th International Wolfram Symposium Presentation Karan Ahuja1, Marjan Vandenabeele2,3, Arefe Nami1, Catherine Verfaillie1, Lieve Moons2, Lies De Groef3 Oligodendrocytes in Wolfram syndrome: bystanders or partners in crime? 1Development and Regeneration Department, KU Leuven Stem Cell Institute, Leuven, Belgium 2Neural Circuit Development and Regeneration Research Group, Biology Department, KU Leuven Brain Institute, Leuven, Belgium […]
8th International Wolfram Symposium Presentation Randal Hand PhD – Director of Neuroscience Research, Prilenia Therapeutics Pridopidine: A selective and potent S1R agonist as a potential treatment for Wolfram Syndrome Points noted: • Pridopidine is currently in clinical trials for neurodegenerative conditions (e.g. ALS (Ph II moving into Ph III); Huntington’s (Ph III). • […]
8th International Wolfram Symposium Presentation Dr Xuehao Cui – PhD student and research fellow University of Cambridge and Moorfields Eye Hospital Exploring novel ocular biomarkers of disease in Wolfram syndrome Abstract: Visual loss in Wolfram syndrome arises primarily due to the selective loss of retinal ganglion cells resulting in optic nerve degeneration. We […]
8th International Wolfram Symposium Presentation Prof Gil Leibowitz – President of the Israel Endocrine Society (IES), Hadassah Medical center, the Hebrew University, Jerusalem, Israel. Pathophysiology and treatment of type 2 Wolfram syndrome. Abstract: Type 2 Wolfram syndrome results from a missense mutation in the CISD2 gene, encoding NAF-1, which transfers Fe-S clusters from the mitochondria […]
Characterization of Mouse Models for Optic Atrophy in Wolfram Syndrome -Urano Lab Venu Gurram, William An, Shrini Bimal, Nila Palaniappan, Toko Campbell, Pooja Neerumalla, Devynn Hummel, Brianna Carman, Cris Brown, and Fumihiko Urano
8th International Wolfram Symposium Presentation Dra. Gema Esteban- Bueno, Dr. Juan R. Coca, Dr. Nicolas Fernández-Fernández, Leticia Fernandez Amores, Miguel Navarro Cabrero, Dra. Aida Berenguel-Hernández and Spanish Multidisciplinary Wolfram Syndrome Group. Spain. Descriptive analysis of 68 patients with Wolfram syndrome with emphasis on sensorineural involvement and possible phenotype-genotype correlation. Abstract: Our work consists in providing […]
8th International Wolfram Symposium Presentation Dr. Vania Broccoli – CNR – National Research Council Institute of Neuroscience, Milan. Italy. New function of Wolframin in regulating the monocarboxylate transporter 1 (MCT1) in glial cells in brain and retina. Abstract: A key pathological manifestation in Wolfram syndrome is the progressive optic atrophy which leads to relentless visual loss. […]
8th International Wolfram Symposium Presentation by Dr Malgorzata Zatyka – Institute of Cancer and Genomic Sciences, University of Birmingham, Edgbaston, Birmingham. UK Depletion of WFS1 compromises mitochondrial function in hiPSC-derived neuronal models of Wolfram syndrome. Abstract: Mitochondrial dysfunction involving mitochondria-associated ER membrane (MAM) dysregulation is implicated in the pathogenesis of late-onset neurodegenerative diseases, but understanding […]
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