Web26 jul. 2024 · Ionis is leading the way in treating root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's and Parkinson's as well as rare diseases like amyotrophic lateral sclerosis (ALS) and Alexander disease. WebAbstract Objective: Alexander disease is a fatal leukodystrophy caused by autosomal dominant gain-of-function mutations in the gene for glial fibrillary acidic protein (GFAP), an intermediate filament protein primarily expressed in astrocytes of …
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Web21 apr. 2024 · April 21, 2024. Alexander disease, Leukodystrophy. In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. (“Ionis”) shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently, treatments are centered around symptom reduction. Web7 aug. 2014 · The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) ... Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, ... Hyperlipoproteinemia Type I Syndrome Disease Pathologic Processes Lipid Metabolism, …
Web2 sep. 2024 · Summary. Hereditary angioedema is characterized by recurrent and unpredictable episodes of subcutaneous and mucosal swelling that can be life threatening. IONIS-PKK-L Rx is a ligand-conjugated ... Web20 jun. 2024 · Background: Diacylglycerol-O-acyltransferase 2 (DGAT2) is one of two enzyme isoforms that catalyse the final step in the synthesis of triglycerides. IONIS-DGAT2 Rx is an antisense oligonucleotide inhibitor of DGAT2 that is under clinical investigation for the treatment of non-alcoholic fatty liver disease (NAFLD) and non-alcoholic …
Web25 jan. 2024 · “This is the first targeted therapy for Alexander disease, and the idea that we soon might be able to address this gene defect is very exciting,” said Florian Eichler, MD, assistant professor of neurology at Harvard Medical School and director of the leukodystrophy service at Massachusetts General Hospital, who was not involved with … Web26 jul. 2024 · CAMBRIDGE, Mass. and CARLSBAD, Calif., July 26, 2024/ PRNewswire/-- Biogen Inc. and Ionis Pharmaceuticals, Inc. announced today that topline data from a Phase 1 b placebo-controlled, multiple ...
Web20 apr. 2024 · Ionis is leading the way in treating the root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's and Parkinson's as well as rare diseases like amyotrophic lateral sclerosis (ALS) and Alexander disease. Ionis' marketed neurological disease medicines include SPINRAZA ®, the …
Web24 dec. 2024 · Over the years, centrally administered ASOs have demonstrated widespread CNS tissue distribution, activity, and substantial phenotypic prevention and/or reversal in mouse models of Huntington's , SMA , Frontotemporal dementia , Parkinson's , TDP-43- and C9orf72-linked ALS , Alexander disease , Pelizaeus-Merzbacher disease , Prion … how far is earth from the sun in metersWeb5 apr. 2024 · Ionis is leading the way in treating the root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's and Parkinson's as well as rare diseases like amyotrophic lateral sclerosis (ALS) and … how far is earth from the sun nasaWebIonis Pharmaceuticals, Inc.’s Post Ionis Pharmaceuticals, Inc. 39,136 followers 2y Edited Report this post ... higgs v foster 2004 case summaryWeb19 aug. 2024 · Ionis previously developed a drug to successfully treat SMA, a neuro-muscular disorder, and they have developed a new drug, ION373, for AxD, that uses the same intrathecal delivery method. ... Study #1 (Outcome Metrics in Alexander Disease) Requires a visit to the CHOP. higgs v farmor\u0027s school 2023Web21 mrt. 2016 · The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain … higgs v farmors school update 2022Web17 nov. 2024 · Alexander disease is a progressive and rare neurological disorder with no cure or standard course of treatment. But a new study led by researchers at the University of Wisconsin–Madison involving a rat model of the disease offers a potential treatment for the typically fatal condition. higgs v farmor\\u0027s schoolWeb17 okt. 2024 · Ionis is leading the way in treating root causes of many neurological diseases and developing antisense medicines for common diseases like Alzheimer's … higgs v farmor\\u0027s school 2023