Ionis alexander disease
Web20 apr. 2024 · Ionis Pharmaceuticals (NASDAQ:IONS) announced the initiation of the pivotal Phase 2/3 study for ION373 in patients with Alexander disease, a rare and generally fatal neurological disorder. The ... Web20 apr. 2024 · Alexander disease is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that protects a nerve fiber and supports...
Ionis alexander disease
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WebTo evaluate safety and efficacy of ION373 in Alexander Disease. Research type. Research Study. Full title. A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, ... Ionis Pharmaceuticals, Inc. Eudract number. 2024-000976-40. Duration of Study in the UK. 4 years, 6 months, 0 days. Web31 dec. 2024 · Alexander disease affects approximately 500 people in the EU. It is caused by a mutation in a protein that creates overproduction of glial fibrillary acidic protein (GFAP) in the brain. ION373 is designed to stop the mutated gene from producing excess GFAP.
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 … WebIn each episode of Ionis Innovations podcast, our host Dave Ecker and his guests will explore the fascinating field of drug discovery. Skip ... she leads research on investigational medicines to treat Alexander disease and Pelizaeus-Merzbacher disease, two rare neurodevelopmental disorders affecting young children. Elaine Pirie, Ph.D ...
Web10 mrt. 2024 · Originator Ionis Pharmaceuticals. Class Antisense oligonucleotides. Mechanism of Action Glial fibrillary acidic protein expression inhibitors; RNA interference. Orphan Drug Status. Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases. Yes - Alexander disease. Web2 okt. 2024 · Trades from $ 1. Ionis Pharmaceuticals, Inc. (. IONS Quick Quote. IONS - Free Report) announced that the FDA has granted an orphan drug designation to its investigational pipeline candidate ION373 ...
Web5 apr. 2024 · Advancement of ION363 to a pivotal trial is the latest example of the power of Ionis' antisense technology to potentially target the root causes of neurological diseases," said C. Frank Bennett ...
Web17 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. Albee Messing, VMD, PhD population of matheson ontarioWeb25 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 … population of mass 2022Web19 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. sharm el sheikh to alexandria flightsWeb30 sep. 2024 · Alexander disease (AxD) is a rare neurological condition characterized as a leukodystrophy, or a disease affecting the myelin sheath (the fatty insulation that protects a nerve fiber and supports signal conduction). Two major types of AxD … population of masset bcWeb30 sep. 2024 · CARLSBAD, Calif., Sept. 30, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, announced today... population of mathura districtWeb10 mrt. 2024 · Phase II/III Alexander disease Most Recent Events 22 Apr 2024 Phase-II/III clinical trials in Alexander-disease (In children, In adults, In infants, In the elderly, In … sharm el sheikh to milanWeb1 jan. 2024 · Deep reductions may keep the disease from progressing and, in the best-case scenario, even reverse progression. For those facing a debilitating disease and early death, the benefits may easily outweigh any potential risks. Ionis has successfully created an ASO that treats Alexander disease in a rat model which mimics the disease features. population of mati davao oriental