Did you know The Orphan Disease Center (ODC, a center within the Perelman School of Medicine at the University of Pennsylvania) has a program to assist patient groups and families in the rare disease community accelerate research for their conditions?
A rare disease diagnosis is often an overwhelming experience. Unfortunately, the field lacks the same knowledge and treatments that are available for more common disorders. The good news is, developing connections with other patients, caregivers, and families can be comforting and invaluable to feeling less alone. The ODC’s JumpStart program is a resource developed to assist individuals and groups of rare disease patients to determine the best path for research in their disease, and to find and develop external resources to support them in reaching those goals. JumpStart’s goal is to effectively assist patient groups to accelerate the research effort in a rigorous and efficient manner.
Generating interest from scientists and clinicians for rare conditions is often a real challenge, especially if research programs did not already exist; this happens when research on a gene or process was ongoing before the disease was identified due to the importance of the gene. The JumpStart program connects patients and foundations to researchers and key opinion leaders and will introduce researchers to a disease state and encourage scientific collaboration. In conjunction with disease/gene-specific experts, the JumpStart program can provide guidance on developing research priorities. JumpStart can even facilitate the establishment of cell lines and the development of animal models for rare disorders.
The ODC will also provide essential guidance on clinical development programs including registry and natural history development. The ODC hopes to ease the burden of rare disease families who are already doing so much to care for their loved ones, organize their communities, and are often fundraising and researching how treatments or cures could be pursued. There are many options in the registry platform space, and the ODC can help to navigate some of the choices.
While JumpStart unfortunately cannot provide financial support, it does offer assistance with the research grant process and can advise on the allocation of funds from fundraising efforts to support the advancement of rare disease research.
If you would like to learn more about how JumpStart could help progress research for your disease, please visit their website here.