Ionis alexander disease
Web29 mrt. 2024 · Ionis is discovering and developing potential treatments for many neurological diseases for which there are few or no disease modifying treatments, including common diseases like Alzheimer's and Parkinson's as well as rare diseases such as amyotrophic lateral sclerosis (ALS) and Alexander disease. Web28 apr. 2024 · Ionis has trials ongoing of multiple other CNS ASOs in symptomatic populations, including those targeting MAPT in PSP ( NCT04539041) FUS in ALS ( NCT04768972) and GFAP in Alexander disease ( NCT04849741 ), plus most importantly, tofersen for SOD1 ALS, which will read out late this summer ( NCT02623699 ).
Ionis alexander disease
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Web17 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 … 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.
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. Web10 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 …
WebIonis Pharmaceuticals announced it has initiated a pivotal clinical study of ION373 in patients with Alexander disease, a rare and generally fatal neurological disorder. … 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.
WebTo determine if the pharmacokinetics and pharmacodynamics of gapmer antisense oligonucleotides (ASOs), containing phosphorothioate backbones and 2'-O-methoxyethyl RNA modifications (2'-MOE ASOs), can be altered by renal disease, a series of experiments were performed in models of chronic kidney disease (CKD) and acute kidney injury (AKI).
Web19 apr. 2024 · A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD) The safety and scientific validity of this study is the … flight ua919Web29 mrt. 2024 · In addition to Alzheimer's disease, our clinical stage neurology programs include ATTR polyneuropathy, ALS, Alexander disease, Parkinson's disease and Angelman syndrome." The Phase 1b trial and its open-label long-term extension (LTE) were designed to evaluate the safety and tolerability of multiple dose levels of IONIS-MAPT Rx … flight ua921Web21 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. flight ua 9066 seat mapWeb29 okt. 2024 · Ionis recently launched a phase III trial of its FUS-lowering ASO jacifusen in 64 patients with confirmed FUS mutations. FUS mutations account for 1–5% of familial ALS, again likely via toxic ... flight ua923Web17 nov. 2024 · Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment Sci Transl Med. 2024 Nov 17;13 (620):eabg4711. doi: 10.1126/scitranslmed.abg4711. … flight ua925WebAxD is a rare, progressive and fatal neurological disease that affects the myelin sheath which protects nerve fibers. AxD is caused by a gain-of-function mutation in the GFAP … flight ua 927Web5 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 ... flight ua936