Huntington’s disease is a neurodegenerative disorder caused by a mutation in the HTT gene. This gene provides instructions for making a protein called huntingtin. Huntingtin is involved in the development and function of neurons in the brain. The HTT gene mutation causes an abnormal expansion of a section of DNA called CAG repeats. This expansion leads to the production of an abnormally long huntingtin protein. The abnormal huntingtin protein interferes with the normal functioning of neurons and causes the death of these cells.
Symptoms of Huntington’s disease typically begin in adulthood and worsen over time. They include involuntary movements called chorea, cognitive decline, and emotional disturbances. Huntington’s disease is diagnosed based on the presence of the HTT gene mutation and a family history of the disorder. There is no cure for Huntington’s disease, and treatments are focused on relieving symptoms.
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Key Trends
In the past decade, there have been significant advances in our understanding of Huntington’s disease (HD), paving the way for new treatments. The HD market is currently small, but is expected to grow as more treatments are approved and awareness of the disease increases.
The most notable recent development in HD is the approval of the first ever therapeutic drug, Spinraza (nusinersen), in December 2016. Spinraza is an antisense oligonucleotide that targets the mutated huntingtin protein and has been shown to improve motor function and delay disease progression in clinical trials. This is a major breakthrough for HD patients and their families, and is expected to drive growth in the HD market in the coming years.
Other key trends in the HD market include:
- Increasing awareness of the disease. In the past, HD was often misdiagnosed or not diagnosed at all, due to its similarity to other neurological conditions.
- Improved diagnosis and treatment options. In addition to Spinraza, there are a number of other promising HD treatments in development, including small molecule drugs, gene therapy, and stem cell therapy. These new treatments are expected to improve HD patient’s quality of life and extend their life expectancy.
- Growing prevalence of HD. HD is a progressive, degenerative disease with no cure. The number of people affected by HD is expected to increase as the population ages and the average life expectancy increases.
- Increasing costs of HD care. HD is a complex disease that requires lifelong treatment. The costs of HD care are expected to increase as more treatments are approved and the number of people affected by HD grows.
Key Drivers
There are a number of key drivers of the Huntington’s disease market.
Firstly, the increasing prevalence of the condition is a key driver. Huntington’s disease is a progressive neurodegenerative disorder that typically affects people in their 30s or 40s. The condition is characterized by a number of symptoms including involuntary movements, cognitive decline, and psychiatric problems. The prevalence of Huntington’s disease is thought to be around 5-10 per 100,000 people.
Secondly, the development of new treatments is a key driver of the Huntington’s disease market. There are a number of new treatments in development that are showing promise in clinical trials. These new treatments are aimed at slowing the progression of the disease and improving the quality of life for patients.
Thirdly, the increasing awareness of the condition is a key driver of the Huntington’s disease market. The condition is often under-diagnosed and many people are not aware that they may be at risk of developing the disease. The increasing awareness of the condition is leading to more people being diagnosed and treated.
Fourthly, the availability of government funding is a key driver of the Huntington’s disease market. In the United States, the National Institutes of Health (NIH) is the main source of funding for research into Huntington’s disease. The NIH provides funding for a number of clinical trials and research projects.
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Market Segments
By Treatment
- Symptomatic treatment
- Disease-Modifying Therapies
Key Players
- AbbVie
- Acorda Therapeutics
- Amgen
- BioMarin Pharmaceutical
- F. Hoffmann-La Roche
- Ionis Pharmaceuticals
- Janssen Pharmaceuticals
- Pfizer
- Sanofi
- Shire
- Teva Pharmaceuticals
- UCB
- Voyager Therapeutics
- Arena Pharmaceuticals
- Axovant Sciences
- Cytokinetics
- Gentium SpA
- Impax Laboratories
- Sunovion Pharmaceuticals
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