Entries by The Snow Foundation

8th International Wolfram Symposium Presentation Liiv M1, Vaarmann A1, Kuum M1, Gupta-Blixt R1, Janickova L1, Hodurova Z1, Cagalinec M1, Zeb A1, Choubey V1, Hickey MA1, Safiulina D1, Yi-Long H2, Gogichaisvili N1, Mandel M1, Plaas M1, Vasar E1, Loncke J3, Vervliet T3, Tsai T-F2, Bultynck G3, Veksler V4, Kaasik A1.1Departments of Pharmacology and Physiology, University of Tartu, Estonia. 2Department of Life Sciences, National Yang Ming Chiao Tung University, Taiwan 3Laboratory of Molecular and Cellular Signaling, KU Leuven, Belgium. 4INSERM UMR-S 1180, University Paris-Saclay, France.

8th International Wolfram Symposium Presentation Liiv M1, Vaarmann A1, Kuum M1, Gupta-Blixt R1, Janickova L1, Hodurova Z1, Cagalinec M1, Zeb A1, Choubey V1, Hickey MA1, Safiulina D1, Yi-Long H2, Gogichaisvili N1, Mandel M1, Plaas M1, Vasar E1, Loncke J3, Vervliet T3, Tsai T-F2, Bultynck G3, Veksler V4, Kaasik A1.1Departments of Pharmacology and Physiology, University of […]

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8th International Wolfram Symposium Presentation Fumihiko Urano, MD, PhD, Samuel E. Schechter Professor of Medicine, Washington University School of Medicine, St. Louis, USA

8th International Wolfram Symposium Presentation Fumihiko Urano, MD, PhD, Samuel E. Schechter Professor of Medicine, Washington University School of Medicine, St. Louis, USA   Novel Therapies for Wolfram Syndrome   Abstract: Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe […]

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8th International Wolfram Symposium Presentation Prof Timothy Barrett, Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham UK, on behalf of the TREATWOLFRAM investigators (Dr Renuka Dias, Dr Ben Wright (UK), Dr Gema Esteban Bueno (Spain), Prof Wojciech Mlynarski (Poland), Dr Christophe Orssaud, Prof Agathe Roubertie (France)

8th International Wolfram Symposium Presentation Prof Timothy Barrett, Institute of Cancer and Genomic Sciences, University of Birmingham, Birmingham UK, on behalf of the TREATWOLFRAM investigators (Dr Renuka Dias, Dr Ben Wright (UK), Dr Gema Esteban Bueno (Spain), Prof Wojciech Mlynarski (Poland), Dr Christophe Orssaud, Prof Agathe Roubertie (France)   Sodium Valproate efficacy and safety in […]

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8th International Wolfram Symposium Presentation Dr Christophe Orssaud – Functional Unit of Ophthalmology, Paris, France

8th International Wolfram Symposium Presentation Dr Christophe Orssaud – Functional Unit of Ophthalmology, Paris, France Audiowolf : Description, contribution and interest of this protocol Alongside the Treatwolfram protocol initiated by Pr T Barrett, a second new protocol has been initiated in France, called Audiowolf. These two protocols are similar in terms of their duration (3 […]

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8th International Wolfram Symposium Presentation Prof Catherine Verfaillie – Stem Cell Institute, KU Leuven, Belgium

8th International Wolfram Symposium Presentation Prof Catherine Verfaillie – Stem Cell Institute, KU Leuven, Belgium   iPSC-based modeling of Wolfram Syndrome and therapeutic genome editing approaches   Points noted: •  Developed iPSC-based models of different cell types – neural cells, ganglion, astrocytes, and blood brain barrier endothelial cells. •  These cell lines can be shared […]

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8th International Wolfram Symposium Presentation Mathias Leinders – Amylyx Pharmaceuticals 

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 […]

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8th International Wolfram Symposium Presentation Dr Benjamin Delprat – University of Montpellier, France

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 […]

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8th International Wolfram Symposium Presentation Karan Ahuja, Marjan Vandenabeele, Arefe Nami, Catherine Verfaillie, Lieve Moons, Lies De Groef

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
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8th International Wolfram Symposium Presentation Randal Hand PhD – Director of Neuroscience Research, Prilenia Therapeutics 

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). • […]

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8th International Wolfram Symposium Presentation Dr Xuehao Cui – PhD student and research fellow University of Cambridge and Moorfields Eye Hospital

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 […]

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