Strategic Plan for Science

GOAL:

To support the development of effective therapeutic treatments through scientific research, improve the quality of clinical research and care available to families, and improve communications and collaborations in PMS research.

OBJECTIVES:

  • To enhance financial resources for PMS research through a coordinated funding strategy
  • To fund one or more grants that increase replication and validation of model system findings
  • To announce one or more funding opportunities per year that build the field of research
  • To fund one or more seed grants per year that facilitate novel directions in PMS research
  • To make patient-contributed data available to explore natural history and enroll patients into clinical research studies & trials.
  • To provide grants that improve clinical trials readiness.
  • To fund one or more seed grants that address a high priority quality of life(QOL) issue in PMS
  • To improve clinical care recommendations through the sponsorship of a consensus meeting.
  • To increase the uptake of clinical care guidelines through the development of content.
  • To improve patients’ access to care through the establishment of PMS Centers of Excellence.
  • To fund at least one grant to understand what factors underlie the variability in PMS.
  • To enable sharing of unpublished data and opportunities for collaboration by holding at least one PMSF-sponsored research meeting per year.
  • To influence PMS research and stimulate new ideas and projects through ongoing communication with scientific stakeholders.
PMSF | Phelan-McDermid Syndrome Foundation