Rare Disease Day: Inspired to develop new treatments for patients


Today is Rare Disease Day and the rare disease community is marking the day by inviting everyone to “join together for better care.” The rare disease community is unique and a very active partner in all aspects of healthcare. Community members openly share information about their conditions to generate awareness. They also work closely with the pharmaceutical industry and academia to develop unique therapies and actively promote transparency in research and education to benefit everyone. We have a lot in common!

A few years ago I had the privilege of attending a clinic for children suffering from a rare disease. I was grateful for the invitation and very moved by their openness to share their experiences with me. One incident from that day has stuck with me, motivating me to do more to help the patients we serve. I saw an 8-year-old boy learning how to self-infuse his medicine. I asked the boy why he wanted to learn to how to give himself a shot at such a young age. His answer was quick:  “If can do it myself, I could help my mom in the morning when she’s so busy.” The 8-year-old’s desire to make it easier for his mom inspired me. It made me recognize that I needed to work harder to develop multiple new approaches to help patients with the issues they face.

The challenges that the rare disease community face provide us with opportunities to develop new therapies, education programs, and diagnostic tools for quicker diagnosis to help make a difference for patients.

I joined GSK less than a year ago because of its values and dedication to build comprehensive therapies for rare diseases. We have a robust pipeline and a dedicated and talented team around the world. We’ve had our share of triumphs, but we’ve also had disappointments, when some of our therapies failed in clinical trials and could not get to patients who needed them. Even with these challenges, the team has worked hard to live by our values and make information available to researchers and patients who need it most.

Please join me and the Rare Disease team in celebrating this day when we pledge to work together with the Rare Disease community to develop treatments for patients with rare diseases.