RASOPATHIES NETWORK USA

Goals:

To build and cultivate a community of practice around RASopathies research, including:

1 Convening bi-annual symposia to bring families, clinicians and researchers together
2 Developing a discussion forum for professionals to foster collaborative research efforts
3 To develop and maintain family-friendly informational resources, including a bibliography for lay and professional audiences
4 Advancing research of the RASopathies - including Costello, CFC, Noonan, Neurofibromatosis type 1, LEOPARD/NSML, toward finding a cure.

1. Convene scientific meetings, which will include inviting family support groups for multiple RASopathy syndromes, in a meaningful way for families, clinicians, researchers and pharmaceutical industry representatives.
2. Expand a website that represents a major resource on RASopathy syndromes for families, clinicians, researchers and the pharmaceutical industry.
3. Strive to stay on the cutting edge of family and patient driven research.
4. Identify partners to support research on the RASopathy syndromes.

1. Ability to connect families, clinicians and researchers around the subject of the RASopathy syndromes.
2. Ability to raise funds adequate to meet our goals.
3. Ability to identify and collaborate with research-funding partners to provide grants to encourage research that benefits all the RASopathies.
4. Ability to continue to raise awareness leading to research of the Rasopathy syndromes.

To date in 2014, RASopathies Network USA:
1. Raised funds for a scientific research grant hosted and managed by the University of Pennsylvania’s CODRT (Center for Orphan Drug Research and Therapy)
2. Restructured the Scientific Advisory Board
3. Taken the lead in the planning and funding of the 4th International RASopathies Symposium (July 2015) including its first NIH R13 Research Meeting grant application
4. Participated in Rare Disease Day on Capitol Hill, In District Lobby Days and spoke at New Jersey State House to educate the government of the importance of NIH funding to advance rare disease research.
5. Invited to participate in the 3rd and 4th World Orphan Drug Congresses in Washington, DC; 3rd Global Genes RARE Patient Advocacy Summits; NORD conference; American Society of Human Genetics Meeting, American Association for Cancer Research Meeting, the European RASopathies Symposia, provided posters at all NCI RASopathies Initiative Symposia.
6. Increased the Board of Directors by two members.
7. Joined the Genetic Alliance, National Organization for Rare Disorders (NORD), Global Genes Foundation Alliance and Corporate Alliance.