Here are the principles that guide our activities and decision making.
Our initial investments are focused on identifying reasonably efficacious repurposed drugs in a timelier manner rather than pouring financial resources and several years into developing a single, potentially highly-effective drug. This affords us successes in each step: minimizing symptoms, halting disease progression, and eventually improving outcomes.
Our primary focus is not patentable novel technologies or molecules. Rather, we are pushing for repurposing approved drugs, using naturally occurring substances, utilizing existing technology, or adapting generics.
As our community of patients is extremely small, clinical trials are not practical. Expanded access, commonly called compassionate use, of existing and experimental drugs is our primary avenue for novel treatments.
For disease progression as rapid as our patients’ experience, time is the most limited resource. Therefore, we are choosing to prioritize speed over financial cost. It does not necessarily mean our organization spends more money. On the contrary, activities that are quick to execute tend to be small and cheap. By choosing to reduce time over money, we not only hope to find treatment faster but also potentially cheaper.
All rare diseases exist in emerging fields of study, SSMD and other GPX4-related diseases included. Instead of trying to prevent failures, we assume failures are inevitable in every activity. We chose to fail fast, fail cheap, maximize the learning from failures, and fail often enough until we learn to do it right.
Openness is about sharing, but also about receiving. We will proactively share everything we create to benefit the community. We will also be open to receiving information that will adapt us and our organization to better reach our mission.