Meeting
Families/Researchers Meeting 2019
The 2019 meeting took place on March 30-31 in Paris (France).
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.
ELA virtuel meeting 2019
Workshop on ALD – AMN
Florian Eichler (United-States) Chair: The Changing World of X-linked Adrenoleukodystrophy
Wolfgang Koelher (Germany): Allogeneic Stem Cell Therapy in Adulthood Cerebral X-ALD
Stephan Kemp (The Netherlands): SCAN study: Screening Adrenoleukodystrophy in the Netherlands Toward newborn screening for ALD
Uwe Meya – Minorix (Spain): MIN-102 for the treatment of X-ALD. Ben Lenail: AMN Clinical Study – A Patient’s Perspective
Workshop on Adult Refsum – Refsum infantile Sauternes – Zellweger – NALD
Bwee Tien Poll-The (The Netherlands): Zellweger spectrum disorders: clinical overview and management approaches
Tanguy Demaret (Belgium): Living-donor liver transplantation in mild Zellweger spectrum disorder, about 3 patients
Workshop on MLD – KRABBE
Caroline Sevin (France) Chair: Symptomatic treatments for MLD and Krabbe’s disease, how to improve day-to-day management
David 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?
David Whiteman (Shire, United-States): Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy
Francesca Fumagalli (Italy): Lentiviral hematopoietic stem cell gene therapy for metachromatic leukodystrophy provides sustained clinical benefit
Nicole Wolf (The Netherlands): Treatment of spasticity in metachromatic leukodystrophy
Shanice Beerepoot (The Netherlands): Peripheral neuropathy in metachromatic leukodystrophy (MLD)
Workshop on MLC – CACH Syndrome – Alexander disease – Canavan St Julien 3 and other vacuolating leukodystrophies
Elena Ambrosini (Italy) Co-chair: Update on MLC research: the advantages of a patient-clinician-researcher network
Jacqueline Trotter (Germany): Understanding the pathology of Vanishing White Matter Disease
Marjo van der Knaap (The Netherlands): A registry for vanishing white matter
Albee Messing (USA): Updates on Research in Alexander Disease
Matthias Klugmann (Australia): Canavan Disease Gene Therapy 2.0
Enrico Bertini (Italy) Co-chair – Mitochondrial Leukodystrophies: a growing list of disorders
Renate J. Verbeek (The Netherlands) : Leukoencephalopathy with loss of white matter – Register
Workshop on PMD – PMD-like (MCT8) – Spastic paraplegia type 2 St Emilion 1 & 2 Pol R III and other hypomyelinating leukodystrophies
Nicole Wolf (The Netherlands): PLP1 mutations in females
Paul Tesar (United-States): Emerging therapeutic research for PMD
Geneviève Bernard (Canada) Chair: RNA polymerase III-induced leukodystrophy: research update.
Workshop on AGS
Yanick Crow (United-Kingdom – France) Chair: Aicardi-Goutières syndrome – an update
Adeline Vanderver (United-States): Update on natural history and Janus Kinase inhibitors in AGS
Workshop on genetic and undetermined leukodystrophies (LDI)
Nathalie Cartier (France) Chair – Genetic and undetermined leukodystrophies
LEUCONNECT
Lucas Alba– Frédérique Le Gourrierec – Sébastien Mariotti – Cindy Oger –Hélène Rochereuil – Elise Saunier Vivar
ELA Infos special n° 106
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