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Dr. Florian Eichler is a Professor of Neurology at\u00a0Massachusetts General Hospital (MGH) and Harvard Medical School. He is also the Katherine B. Sims Chair in Neurogenetics. As\u00a0Director of the Leukodystrophy Service and Director of the Center for Rare Neurological Diseases (CRND) at MGH,\u00a0he\u00a0aims\u00a0to advance care and treatment for\u00a0devastating\u00a0neurogenetic conditions. After completing a neurogenetics fellowship at Johns Hopkins and residency training at MGH, he established a laboratory exploring\u00a0the relationship of mutant genes to specific biochemical defects and neurodegeneration.\u00a0The laboratory\u00a0identified neurotoxic\u00a0desoxysphingoid\u00a0bases that accumulate in mutant transgenic mice and humans with\u00a0hereditary sensory neuropathy type 1 (HSAN1).\u00a0This work\u00a0led to a first clinical trial\u00a0of\u00a0substrate supplementation therapy for patients with\u00a0HSAN1.\u00a0Dr Eichler is\u00a0also\u00a0the Co-PI of\u00a0the\u00a0gene therapy trial of\u00a0adrenoleukodystrophy\u00a0(ALD)\u00a0that reported on first successful outcomes in the New England Journal of Medicine and received FDA approval in September 2022. For this work,\u00a0he\u00a0received the Martin Research Prize from MGH and the Herbert\u00a0Pardes\u00a0Clinical Excellence Award from the Clinical Research Forum.<\/p>\n
He is a\u00a0Co-PI of the\u00a0Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), a consortium of scientists\u00a0working to promote advances in the diagnosis and treatment of leukodystrophies, as well as\u00a0president of the consortium ALD Connect, a patient-powered research network dedicated to curing ALD. He runs several gene therapy trials at MGH, including for Canavan Disease, Tay Sachs and Sandhoff Disease, and Alexander Disease. Recently he became Co-Director of the Precision Therapeutic Unit in the Center for Genomic Medicine at MGH and serves as ad-hoc member on the Advisory Council of NINDS.<\/p>\n","protected":false},"excerpt":{"rendered":"
Dr. Florian Eichler is a Professor of Neurology at\u00a0Massachusetts General Hospital (MGH) and Harvard Medical School. He is also the Katherine B. Sims Chair in Neurogenetics. As\u00a0Director of the Leukodystrophy Service and Director of the Center for Rare Neurological Diseases (CRND) at MGH,\u00a0he\u00a0aims\u00a0to advance care and treatment for\u00a0devastating\u00a0neurogenetic conditions. After completing a neurogenetics fellowship at<\/p>\n","protected":false},"author":6,"featured_media":5609,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[26],"tags":[],"class_list":["post-5608","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-creative"],"aioseo_notices":[],"aioseo_head":"\n\t\t\n\t\n\t\n\t\n\t\n\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t\n\t\t