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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 ]
Huntington's disease, which affects about 30,000 Americans, is a fatal, inherited disorder that causes progressive movement, psychological and cognitive problems. If a parent has it, their ...
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.
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.
In 2017, SOM Biotech entered into an agreement with Nippon Chemiphar (Japan) to collaborate for the SOM3355 program for the treatment of chorea in Huntington's disease. From 2018 to 2019, SOM Biotech successfully performed a clinical proof-of-concept phase IIa study assessing SOM3355 efficacy and safety in reducing chorea in Huntington’s disease.
HOPES Logo. The Huntington's disease Outreach Project for Education at Stanford (HOPES) is a student-run project at Stanford University dedicated to making scientific information about Huntington's disease (HD) more readily accessible to patients and the public.
Originally, the High Q foundation aimed to identify targets for treatments, while CHDI – the 'Cure Huntington's Disease Initiative' – was a sister organization allied to the Hereditary Disease Foundation, that focused on developing drugs to hit those targets. [1] 'CHDI', which is officially no longer an abbreviation for anything, now refers ...
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