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Branaplam (development codes LMI070 and NVS-SM1) is a pyridazine derivative that is being studied as an experimental drug.It was originally developed by Novartis to treat spinal muscular atrophy (SMA); since 2020 it was being developed to treat Huntington's disease but the trial ended in 2023 due to toxicity concerns.
Huntington's disease (HD), also known as Huntington's chorea, is an incurable neurodegenerative disease [7] that is mostly inherited. [8] The earliest symptoms are often subtle problems with mood or mental/psychiatric abilities. [9] [1] A general lack of coordination and an unsteady gait often follow. [2]
The HOPES website provides information about topics including the causes and symptoms of HD, existing drugs and supplements that may help HD patients, recent advances in HD research and lifestyle choices for managing HD. [1]
Prana Biotechnology's Huntington disease drug, PBT2, is safe, but whether it helps patients enough to get approved remains to be seen. The press release headline "Prana Announces Successful Phase ...
Tetrabenazine is a drug for the symptomatic treatment of hyperkinetic movement disorders.It is sold under the brand names Nitoman and Xenazine among others. On August 15, 2008, the U.S. Food and Drug Administration approved the use of tetrabenazine to treat chorea associated with Huntington's disease.
PBT2 is a safe-for-human-use Zinc ionophore [1] and an experimental drug candidate. It is a second-generation 8-hydroxyquinoline analog [2] intended to be a successor to clioquinol and a potential treatment of Alzheimer's disease [3] and Huntington's disease.
The Journal of Huntington ' s Disease is a quarterly peer-reviewed scientific journal in neuroscience that covers all aspects of Huntington's disease and related disorders. It was established in 2012 and is published by IOS Press. The editors-in-chief are Blair Leavitt (University of British Columbia) and Leslie Thompson .
The trial found no difference in treatment effects between AV-101 and placebo. [1] [11] Preclinical studies in animal models suggested efficacy in treating neuropathic pain. [12] AV-101 showed efficacy in an animal model of Huntington's disease [4] and rapid-acting antidepressant effects similar to ketamine in behavioral models of depression in ...