HUNTINGTON DISEASE DRUG PIPELINE

Huntington disease research is constantly adding more potential drug treatments into the pipeline. The clinical phases of research take time and must go through specific phases before it can be prescribed by a physician in an HD clinic. The basic phases are:

Phase 1: Testing a new drug or treatment in a small group of people to determine its safety, safe dosage range, and side effects.
Phase 2: Testing a new drug or treatment in a larger group of people to determine its effectiveness and safety.
Phase 3: Testing a new drug or treatment in a large group to confirm its effectiveness, monitor its side effects, compare it to other treatments, and collect safety information.
Phase 4: After the drug or treatment has been approved and marketed, studies are conducted to gather information on the drug or treatment’s effect on populations and any side effects associated with long-term use.

HD has more drugs in the pipeline than ever before. The graphic below illustrates some of the treatments by their most currently known phase of development.

Credit: HD Insights, Vol. 24

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