IMPORTANT

Before participating in a study, talk to your health care provider and learn about the risks and potential benefits.

This study is to look at whether so-called radioligands (small radioactive particles) can detect mutant Huntingtin (mHTT) in the brain. The imaging study uses PET scans of the brain to see if when the radioligands are present is related with the expression of mHTT in brain cells.  If the relationship is proven correct then it will a valuable way to measure mHTT and track if huntingtin lowering therapies actually lower mHTT in the brain. 

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PRidopidine Outcome On Function in Huntington Disease (PROOF-HD)

PROOF-HD is a Phase 3 clinical study that evaluates the efficacy and safety of pridopidine in patients with early stage of Huntington Disease. Pridopidine is a small molecule developed by Prilenia for the treatment of neurodegenerative disorders such as HD

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The study is a phase 1/2 and will look at the safety and how participants tolerate a new antisense oligonucleotide(ASO) in early stage disease, with a drug called WVE-003.

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The European Phase Ib/II clinical trial of AMT-130 for the treatment of HD will explore the safety, proof of concept, and dosing in 15 total patients with with early manifest Huntington’s disease split into a five person manifest Huntington’s disease split into a five person, low-dose open-label(patients know what they are getting) cohort, followed by a nine patient, higher-dose open-label cohort. All patients will be dosed with AMT-130. The therapeutic goal is to inhibit the production of the mHTT. Using AAV vectors to deliver micro-RNAs directly to the brain. A one time injection.

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This is the first study of the drug branaplam in adults with HD to determine the correct dose required to lower mHTT levels in the CSF to a degree expected to be efficacious over longer periods of time.

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