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Huntington's disease treated for first time (bbc.com)

709 points by _zie · 305 days ago · 121 comments on HN

Article summary

Doctors have successfully treated Huntington's disease for the first time, with a new gene therapy slowing the disease's progression by 75% in patients. The treatment, which involves delicate brain surgery, has given patients decades of 'good quality life' and has the potential to prevent symptoms from emerging if administered early. The disease, which is fatal and has no cure, affects approximately 75,000 people in the UK, US, and Europe. The treatment is expected to be expensive, but it offers hope for those affected by the disease.

Main themes

  • Huntington's disease treatment
  • Gene therapy
  • Brain surgery
  • Medical breakthroughs
  • Funding and research
  • Rare diseases

What commenters say

  • The new treatment offers hope for those affected by Huntington's disease, potentially preventing symptoms from emerging if administered early.
  • The high cost of the treatment may limit its availability to those who need it, highlighting the need for affordable healthcare options.
  • The development of this treatment is a testament to the importance of funding and research in the scientific community, often driven by government agencies and public money.
  • Some argue that the focus on rare diseases like Huntington's is not driven by profit, but rather by the efforts of advocacy and funding groups, while others point out that orphan diseases can be extremely profitable for private firms.
  • The use of gene therapy and brain surgery in this treatment raises questions about the potential risks and benefits of such approaches, including the challenge of delivering the treatment across the blood-brain barrier.
  • The impact of this treatment on the quality of life for patients and their families cannot be overstated, with some commenters sharing personal experiences and expressing gratitude for the potential of this breakthrough.
  • There is a need for more transparency and education about the role of government funding and research in driving medical breakthroughs, as well as the importance of fundamental research in advancing our understanding of diseases like Huntington's.
  • The development of this treatment highlights the complex interplay between public and private funding, research, and healthcare, with some arguing that a more equitable and sustainable model is needed to support medical innovation.