Rare Disease Day 2020
In the spirit of Rare Disease Day, we are both reflecting on the past — in particular, the work we started with milasen two and a half years ago — and are looking toward the future. We are considering the best way to build upon what we learned from milasen, to make a difference for more children and more families.
We believe that continuing work in this space is only worth it if we think big:
How can we advance the tools that make n of 1 treatments possible, so they are accessible to those in need? We need to pursue this in a way that is safe and equitable, considering many complex scientific, clinical, regulatory, and ethical questions.
This is a learning process, but we are fortunate to learn alongside many perseverant, creative, and resilient families, researchers, and other collaborators. With each case, we gain insight that makes the next patient’s journey just a bit easier. Tim had the opportunity to share some of these insights as he discussed milasen and the path forward from here on panels last week at both the FDA and the NIH Rare Disease Day events.
We’re also celebrating that on Saturday, Leiden University Medical Center announced the launch of the Dutch Center for RNA Therapeutics — a center intending to become the EU center for n of 1 rare disease treatment. It is a virtual center where Dutch experts can collaborate to design individualized oligo therapeutics for patients. As Mila’s story has received global press coverage, we have gained a growing group of international collaborators and are excited to see the strides that they make with this work.
