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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 ]
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.
Huntington's disease: HDL2 Huntington's disease–like 2: HELLP syndrome: Hemolytic anemia, elevated liver enzymes and low platelet count syndrome HeV Infection Hendra virus infection HF Heart failure: HFA High-functioning autism: HFMD Hand, foot, and mouth disease: HFRS Hemorrhagic fever with renal syndrome: HI Hearing impaired: HiB disease
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.
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