Several members of the OAA community attended this year’s NORD summit in Washington, DC Oct 19-21. Irfan Patel and his son Yusuf helped recruit volunteers for the RareAction Network, a NORD-led organization to support rare disease advocates with grassroots campaigns for state policies that support the rare disease research and healthcare.
Jana Monaco, OAA board member and mother of two children with isovaleric acidemia, advocated for newborn screening by speaking at a panel session. Jana has also shared her story in the newly published book “Writers for Rare: An Anthology of Personal Stories from the Rare Disease Space”. Kathy Stagni, executive director of OAA, and Haley Stubbs, research program manager of OAA, attended the patient registry workshop. NORD staff, as well as health economists, and patient advocates from industry shared the importance of registry data in advancing research and clinical trials. Registry survey data can be used to prioritize measuring changes in the most impactful symptoms and can establish baseline data for the typical course of organic acidemias.
We always look forward to the summit. It's a great way to get to know the innovations occurring in the rare disease space. Moreover the networking opportunities make you feel that you are not alone in this journey
