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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]
AB-1001 is an experimental gene therapy developed for Huntington's disease. It is intended to increase the production of the CYP46A1 enzyme and cholesterol levels in the brain. [ 1 ] [ 2 ]
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.
The group's ultimate goal is to find new therapies for Parkinson's disease and Huntington's disease. These disorders have in common that they are caused by disturbances in the part of the brain called the basal ganglia. To achieve this goal, the group wants to establish and develop a strong, multidisciplinary research environment.
PBT2-201 (EURO) was a 12-week randomized, double-blind, placebo-controlled, parallel three-group study (Phase II) to assess the safety, tolerability and efficacy of two dose levels of PBT2 to slow progression of disease in patients with early AD.
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