Web11 dec. 2024 · Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain. As the phase 1/2a trial progressed, the dose of IONIS-HTTRx was increased several times according to the ascending-dose trial design. Web8 apr. 2024 · Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. In December 2024, Roche licensed the investigational molecule from Ionis Pharmaceuticals.
Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner Following ...
WebIONIS-HTTRx is designed to reduce the production of all forms of the huntingtin (HTT) protein, which in its mutated variant (mHTT) is responsible for HD. As such, IONIS … Web11 dec. 2024 · Meanwhile, Ionis announced in June that all patients in the completed trial would be offered a place in an open-label extension to receive IONIS-HTTRx. The results of the trial and plans for the ongoing IONIS-HTTRx programme will be presented in detail at forthcoming scientific meetings and prepared for peer-reviewed publication. florida children\u0027s and family
Therapeutic Update on Huntington’s Disease: Symptomatic Treatments …
WebCirculation:I型糖尿病患者易患自体免疫相关心脏衰竭 1型糖尿病患者,尤其是血糖控制不好的患者,患心血管疾病的风险比一般人群明显要高。更令人费解的是,在1型糖尿病患者中,许多心血管疾病的危险因素与2型糖尿病相关的已知危险因素并不一致。 Web7 mei 2024 · An international team of researchers has conducted a randomized, double blind, Phase I-IIA clinical trial for the use of IONIS-HTTRx in the treatment of Huntington’s disease. The main aim of the paper, published in the New England Journal of Medicine, was to determine whether the drug was safe for use in people with Huntington’s disease. WebSince 2013, when Ionis and Roche started their alliance, the teams in both companies have collaborated closely in advancing the clinical development of IONIS-HTTRx. Going forward, Roche will now become solely responsible for the further clinical development including trials to demonstrate the safety and efficacy of IONIS-HTTRx. florida children\u0027s theatre