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2017 - 2020 Strategic Plan

PRIORITY 1 – Expand science and medicine program to further encourage finding treatments or a cure

Outcome 1.1 – Facilitate pre-clinical and clinical studies/trials

  • Expedite efforts in TAZPOWER trial
  • Initiate bezafibrate clinical trial
  • Prepare for gene therapy clinical trial
  • Advance the understanding of BTHS (natural history study/aging)
  • Continue to explore development of enzyme replacement therapy
  • Continue to explore development of repurposing drugs and/or screening compounds for potential therapy
  • Continue to explore development of lipid replacement therapy
  • Encourage the publication of amino acid supplementation in BTHS
  • Develop a scientific/medical framework to outline advances in BTHS

Outcome 1.2 – Support researchers through research grant program

  • Increase the number of qualified research grant applications by 10% each year
  • Continue to support BTHS publications through open access
  • Establish research priorities to advance drug discovery

Outcome 1.3 – Establish next iteration of Barth Syndrome Registry and Repository (BRR2.0)

  • Where possible, coordinate validated questions and responses with other registry groups relevant to BSF
  • Use BRR2.0 to support research and clinical trials
  • Increase enrollment in BRR2.0 each year
  • Promote and track usage by researchers and physicians
  • Work toward BRR 3.0
    • Establish a BRR advisory committee and strategic plan for deliverables for the BRR
    • Explore development of shared institutional partnership agreements to connect data from 1.0 and 2.0, genetic variant database and biological specimens

Outcome 1.4 – Identify high priority areas for focused research Investigate benefits of genomic sequencing

  • Investigate benefits of mRNA sequencing with various pharmaceutical compounds

PRIORITY 2 – Enhance the capacity of Barth Syndrome Foundation to better meet its mission

Outcome 2.1 – Ensure that the Barth community is prepared to participate in clinical trials

  • Develop clinical trial educational materials to foster a culture of engagement and participation in clinical trials for the development of therapies
  • Establish clinical trial readiness framework

Outcome 2.2 – Increase the breadth and depth of committed contributors

  • Increase contributions from new sources
  • Increase donor retention rate year-to-year (excluding memorials/honorariums)
  • Encourage repeat contributors to increase their donation
  • Increase number of solicitors and campaign events
  • Encourage a culture of philanthropy throughout the organization
  • Further diversify the BSF Board of Directors
  • Better capitalize on Board members’ abilities to contribute to BSF

Outcome 2.3 – Increase the number of identified individuals with BTHS

  • Expand reach through connection with all diagnostic laboratories
  • Focus attention on pediatric cardiologists
  • Encourage those diagnosed with BTHS to affiliate with BSF

Outcome 2.4 – Partner with institutions and create alliances that accelerate research, increase awareness, and improve treatment

  • Track non-BSF funding for Barth syndrome-specific research and/or clinical trials
  • Deepen strategic alliances with other agencies that will propel BSF’s mission
  • Explore partnering with for-profit entities
  • Maintain ongoing communication with government, umbrella and peer partner agencies
  • Identify pertinent educational meetings/conferences for BSF representatives to further the overarching mission

Outcome 2.5 – Integrate affiliates as appropriate

  • Expand financial resources for BTHS research through affiliate coordinated funding strategy
  • Encourage participation and leadership by affiliates in BSF programs
  • Expand reach by establishing new affiliates
  • Encourage sharing of best practices and experiences among affiliates

Outcome 2.6 – Facilitate participation at centers of excellence/clinics for BTHS

  • Maximize the number of families who participate by providing information
  • Encourage collaboration among centers/clinics and participants’ local physicians
  • Encourage clinicians at centers/clinics to assist in the development of treatment guidelines

Outcome 2.7 – Evaluate viability of venture philanthropy

PRIORITY 3 – Build confidence and trust in Barth Syndrome Foundation’s community and resources

Outcome 3.1 – Sustain a strong, mutually supportive community with a shared mission

  • Maximize family participation/engagement in the following areas:
    • Sci/Med – BRR2.0, clinical trials, centers of excellence/clinics
    • Fundraising/Volunteerism – time, talent, treasure, testimonials
    • Family Support – Listserv, conference, regional outreach
  • Maximize the number of clinicians and researchers who:
    • Attend the conference o Contribute on the Sci/Med Listserv
    • Request information about BSF; refer patients to BSF
    • Further develop use of social media to promote awareness and extend BSF’s reach

Outcome 3.2 – Be an essential source of credible information for families, clinicians, and researchers

  • Enhance: website, printed publications, Listserv, videos, grand rounds, clinic visits, etc.
  • Engage adults with BTHS in program evolution, including transition to adult care and beyond
  • Engage carriers in program evolution
  • Encourage researcher, clinician, affected individual, and family ambassadors of BSF
  • Facilitate treatment guidelines for BTHS
  • Develop communication strategy to ensure timely and accurate dissemination of information of interest to the BTHS community
  • Develop a replacement for the Listserv that can leverage technology to identify common family needs and provide direction for program priorities
  • Establish a clinical consensus committee to develop/update evidence-based approaches to care (i.e., recommendations, fact sheets and other publications)
  • Promote a culture of advocacy by providing access to credible information about policies and issues that would impact the provision of care for those who have BTHS

Outcome 3.3 – Continue to organize the finest scientific, medical and family biennial conference on BTHS


Last updated 2/28/2019

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