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Tetrabenazine was approved in 2000 for treatment of chorea in Huntington's disease in the EU, and in 2008 in the US. [88] Although other drugs had been used "off label", tetrabenazine was the first approved treatment for Huntington's disease in the U.S. The compound has been known since the 1950s.
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 ...
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 ]
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.
Pharmaceutical firm Roche and biotech company Isis Pharmaceuticals have agreed to a partnership to develop treatments for Huntington's disease. The two companies, which announced the agreement in ...
The drugmaker said liraglutide, a generic of Novo Nordisk's drug Victoza for patients with type 2 diabetes, also received a lot of attention and helped drive growth in the segment.
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.
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 ...
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