Our Commitment to the Rare Disease Community
The ability to help others in need is the reason why I became a physician. And this commitment is unconditional. It does not end because you happen to have a disease that is uncommon and/or misunderstood. In fact, in these scenarios, there is an even greater need to help.
This is why I’m so proud to work at an organization that shares my commitment. Today marks the annual celebration of Rare Disease Day. Observed in more than 100 countries worldwide, the day aims to raise awareness and generate change for the 300 million people worldwide living with a rare disease, their families and caregivers.¹
At Moderna, our mission is to deliver the greatest possible impact to people through mRNA medicines. We measure our success on the progress we make on improving human health. With our mRNA platform, we see an incredible potential, and responsibility, to use the technology we have researched and developed to potentially improve the quality of life for those affected by rare diseases.
We are studying investigational mRNA therapeutic candidates for patients with propionic acidemia (PA), methylmalonic acidemia (MMA), and glycogen storage disease type 1a (GSD1a), and we look forward to sharing data when available.
We believe this is just the beginning and that mRNA could potentially help patients with a large variety of protein and enzyme deficiencies. We are committed to advancing mRNA therapeutic candidates to address diseases – whether they affect one person or a million.
We are also incredibly inspired by the stories we have heard from the people who are affected by rare diseases, including families and patient organizations. We will continue to work harder and faster because we know that you are waiting. With our innovative mRNA science and through our deep commitment to working with patient communities, we believe we could change how medicines for rare diseases may be delivered in the future. We will not stop working until we achieve that goal.