Strategic Research Priorities
Values
- Data produced during research should be shared openly to enable collaboration
- Research that is synergistic across the family of MAST genes is preferred over single-gene research
- Patients/caregivers should be directly involved in the research plan
Current Priorities
JumpStart Program with the Orphan Disease Center
We are proud to announce that we have joined the JumpStart program with the Orphan Disease Center. The Orphan Disease center, a center within the Perelman School of Medicine at the University of Pennsylvania, works closely with patient groups and foundations, pharma and biotech, and the academic community to drive therapeutic development for rare diseases.
As part of this program, our first two research goals are:
- Raise $100,000 for potential research grants
- Identify 250 patients for potential future research
Network with other rare disease groups
Create a packet for families
- Resources that can be helpful in the present
- How to contribute to the long-term research plan
Future Research Priorities
Partner with other research organizations
Explore drug repurposing studies
Develop a clinical symptom questionnaire
Determine which symptoms are most challenging to determine which treatments might have the most daily life impact for patients and caregivers.