A Potential Solution for Rare Diseases

Many of us have had a relative or someone immediate in our lives pass away from a rare disease. Rare diseases can be debilitating and sometimes deadly. Since these diseases are rare, we face the frustrating conclusion that no treatment or cure has been developed. But, the future of medicine may be nearing.

I examined New York Times article, “A Battle Plan For a War on Rare Diseases,” which discussed Bertrand, a child born with an imperative genetic disease, and how his father, Dr. Matthew Might, was able to discover therapy for him. He worked on research to find medicine and treatment for a rare mutation that only about 60 other people live with. It took Dr. Might four years to discover what condition his son had, find a treatment, and work with a company to develop a stronger version of the drug.

The idea of personalized treatment opens up a world of possibilities in terms of medicine discovery and efficiency in how it is developed. Dr. Might strongly believes that if more doctors work with families of children who have rare diseases, we will be able to find many treatments and therapies. Sharing information from one case with the world will put us farther down the road of discovery than we have ever been.

Our world has advanced rapidly in the past century and with that comes a longer healthier lifestyle. As we become more developed, we are beginning to discover rare diseases that a very small percent of the population are diagnosed with. With this, we are expecting to find cures to these diseases.

We are running into a problem where it is not common to find treatments for rare diseases because only a small percent of the population have them. People who are close to those with rare diseases such as parents must work together on research to find therapies.

I believe it is not this simple as Dr. Might proposes to complete this task. Scientists work for years trying to make some progress in finding cures for diseases. So what makes a cure for rare disease easier to find especially since the population to test the therapy on is smaller?

Although it seems step by step simple, I believe it would take many years to develop treatments for rare diseases but as long as we work towards it, we can find it.