In the spring of 2013, a survey was circulated to the RDCRN Consortium Principal Investigators (PIs) and Patient Advocacy Group (PAG) representatives of their consortium to determine how PAGs and their affiliated consortia interacted and whether this interaction was interpreted as positive or negative by both PAG representatives and Consortium PIs. A total of 28 PAG representatives, each from a different PAG, and all 17 RDCRN Consortium PIs completed the survey.
RDCRN Investigators and Patient Advocacy Groups – A Partnership
National Organization for Rare Disorders Honors the RDCRN at their Annual 'Partners in Progress' Gala
As an innovative program of the Office of Rare Diseases Research (ORDR) at NIH, the Rare Diseases Clinical Research Network (RDCRN) demonstrates the value of collaborative research networks to accelerate clinical research in rare disorders. By harnessing the combined power of top researchers, affected individuals and advocacy groups, this very successful model for rare disease research engages more than 80 patient groups (representing 172 rare conditions) in more than 100 studies and has resulted in over 700 publications.
Patient Advocate Spotlight: Erica Barnes Fights for Children with Metachromatic Leukodystrophy and Other Rare Diseases
Erica Barnes, MS, CCC-SLP, is the co-founder of Chloe’s Fight Rare Disease Foundation (CFRDF), a patient advocacy group that supports research to find and implement effective cures and treatments for rare childhood genetic diseases. She also serves as a board member of Cure MLD, state ambassador of the Minnesota RareAction Network, and advocacy committee chair of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN). Here, she shares her journey to rare disease advocacy, impactful work by CFRDF, and collaboration with GLIA-CTN.