{"id":20354,"date":"2024-07-02T15:07:11","date_gmt":"2024-07-02T13:07:11","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&p=20354"},"modified":"2024-07-10T11:50:24","modified_gmt":"2024-07-10T09:50:24","slug":"families-researchers-meeting-2019","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/families-researchers-meeting-2019\/","title":{"rendered":"Families\/Researchers Meeting 2019"},"content":{"rendered":"\n\n
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Families\/Researchers Meeting 2019<\/strong><\/h3>\n <\/div>\n
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The 2019 meeting took place on March 30-31 in Paris (France).<\/strong><\/p>\n

Every year, ELA organizes a meeting for patients and their families. Many researchers coming from all around the world present the latest scientific and medical advances and answer the questions of patients and families. This annual event is an exceptional opportunity for patients and their families to interact with scientists specialised in leukodystrophies and myelin diseases and learn from their diseases.<\/p>\n <\/div>\n <\/div>\n<\/section>\n\n\n\n

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ELA virtuel meeting 2019<\/h3>\n
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Workshop on ALD \u2013 AMN
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Florian Eichler (United-States) Chair: The Changing World of X-linked\u00a0Adrenoleukodystrophy
\nWolfgang Koelher (Germany): Allogeneic Stem Cell Therapy in Adulthood Cerebral X-ALD
\nStephan Kemp (The Netherlands): SCAN study: Screening Adrenoleukodystrophy in the Netherlands Toward newborn screening for ALD
\nUwe Meya – Minorix (Spain): MIN-102 for the treatment of X-ALD. Ben Lenail: AMN Clinical Study – A Patient\u2019s Perspective<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on Adult Refsum \u2013 Refsum infantile Sauternes \u2013 Zellweger \u2013 NALD
\n<\/strong>Bwee Tien Poll-The (The Netherlands): Zellweger spectrum disorders: clinical overview and management approaches
\nTanguy Demaret (Belgium): Living-donor liver transplantation in mild Zellweger spectrum disorder, about 3 patients<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on MLD \u2013 KRABBE
\n<\/strong>Caroline Sevin (France) Chair: Symptomatic treatments for MLD and Krabbe’s disease, how to improve day-to-day management
\nDavid Wenger (United-States): Conditions for the bone marrow plus gene therapy treatment of the mouse model of Krabbe disease, too little, too late or just right?
\nDavid Whiteman (Shire, United-States): Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy
\nFrancesca Fumagalli (Italy): Lentiviral hematopoietic stem cell gene therapy for metachromatic leukodystrophy provides sustained clinical benefit
\nNicole Wolf (The Netherlands): Treatment of spasticity in metachromatic leukodystrophy
\nShanice Beerepoot (The Netherlands): Peripheral neuropathy in metachromatic leukodystrophy (MLD)<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on MLC – CACH Syndrome \u2013 Alexander disease \u2013 Canavan St Julien 3 and other vacuolating leukodystrophies
\n<\/strong>Elena Ambrosini (Italy) Co-chair: Update on MLC research: the advantages of a patient-clinician-researcher network
\nJacqueline Trotter (Germany): Understanding the pathology of Vanishing White Matter Disease
\nMarjo van der Knaap (The Netherlands): A registry for vanishing white matter
\nAlbee Messing (USA): Updates on Research in Alexander Disease
\nMatthias Klugmann (Australia): Canavan Disease Gene Therapy 2.0
\nEnrico Bertini (Italy) Co-chair – Mitochondrial Leukodystrophies: a growing list of disorders
\nRenate J. Verbeek (The Netherlands) : Leukoencephalopathy with loss of white matter – Register<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on PMD – PMD-like (MCT8) – Spastic paraplegia type 2 St Emilion 1 & 2 Pol R III and other hypomyelinating leukodystrophies
\n<\/strong>Nicole Wolf (The Netherlands): PLP1 mutations in females
\nPaul Tesar (United-States): Emerging therapeutic research for PMD
\nGenevie\u0300ve Bernard (Canada) Chair: RNA polymerase III-induced leukodystrophy: research update.<\/p>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on AGS
\n<\/strong>Yanick Crow (United-Kingdom – France) Chair: Aicardi-Goutie\u0300res syndrome – an update
\nAdeline Vanderver (United-States): Update on natural history and Janus Kinase inhibitors in AGS<\/p>\n<\/div>\n<\/div>\n<\/div>\n

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Workshop on genetic and undetermined leukodystrophies (LDI)
\n<\/strong>Nathalie Cartier (France) Chair \u2013 Genetic and undetermined leukodystrophies<\/p>\n<\/div>\n<\/div>\n<\/div>\n

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LEUCONNECT
\n<\/strong>Lucas Alba\u2013 Fre\u0301de\u0301rique Le Gourrierec – Se\u0301bastien Mariotti \u2013 Cindy Oger \u2013He\u0301le\u0300ne Rochereuil – Elise Saunier Vivar<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n\n <\/div>\n<\/div>\n\n\n\n\n\n

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ELA Infos<\/strong> special n\u00b0 106<\/h3>\n <\/div>\n
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Download the associated documents<\/h3>\n