After learning of rare medical conditions such as butterfly syndrome and muscular dystrophy, I asked: “Why would researchers invest time and money into solutions that would only benefit a small percentage of human race?”
To this question, my friend answered:
- With finite resources, governments, organisations and companies will only fund what will give valuable returns.
- There may be chance discoveries that lead to other scientific breakthroughs.
After reading the article, “Determined Parents are Moving the Needle on Gene Therapy” by Emily Mullin, there are further answers:
- Gene therapies have become safer and better at hitting intended targets in the body, leading to a handful of remarkable cures in clinical trials. The success rate has risen.
- Hence, advocates for rare-disease patients are increasingly establishing patient advocacy organisations, raising money for research and even founding their own biotechnology startups to find treatments.
- Also, many rare diseases are monogenic – caused by a mutation in one gene. It is significantly easier to engineer a targeted protein to reverse the disease.
If you were a medical researcher, would you invest your entire career on a solution that would only benefit a few hundred persons in the world?