On September 17, the Child Neurology Foundation Team went to The Global Genes Patient Advocacy Summit in San Diego, California. As the largest gathering of rare disease patients, caregivers, thought leaders and other rare disease stakeholders in the world, the RARE Patient Advocacy Summit is an unparalleled opportunity to forge meaningful connections with other rare advocates.
There is a robust intersection between the child neurology and rare disease communities. Of the 7000 rare diseases, over half of them have a neurological component, and 75% of those originate in childhood.
While at Global Genes, the CNF team took the time to meet with advocacy and industry partners to talk about ways to better support families with rare disease, and also engaged with the community via our exhibit booth and events hosted by other attendees.
Having a conversation with #RAREis
“Our aim is to really serve as a collaborative center of education and support for children and families. Many families that are part of the rare disease community are also part of the child neurology community. We want them to know that there is a robust network of resources and support, because we really, really want families to not feel isolated and like they’re walking alone.”
Brin continued, “Collaboration is at the heart of everything we do; we especially try to manifest that in the rare disease community. We believe you can go further, if you go together. There’s great value in the rare disease community, advocating for single disease awareness. And we definitely recognize the importance of those communities feeling heard and seen.”
The full interview will be posted in the coming weeks on the RAREis Community blog.
Connecting with our community
Over our three days in the exhibit pavilion, we reached out to patients, families and providers with lived experience and professional expertise in the patient journey to find out, what is the most important conversation we are not having in the child neurology community?
We were overwhelmed by the insight, stories and expertise that were shared with us. Thank you to all of the patients, advocates and industry leaders that stopped by our booth to share your stories.
“We still don’t understand all the fundamentals,” said Michael Jolly of Premier Research, a contract research organization that runs clinical studies for a wide range of pharmaceutical companies. “We need to get a handle on basic research and look at the questions that are being asked so we can achieve better and more conclusive research for our patients.”
“Consistency is medical treatment has been tricky for me—the same treatment procedure in the same hospital can be completely different,” says Madison McLaughlin, who is one of three siblings in the McLaughlin family who has a rare disease. “Working with one physician and taking the time to find out what works for me only to have a completely different, and sometimes less effective, experience somewhere else can be frustrating.
Here are some of the other responses that we received during our time in San Diego.
Some of the many responses we received while at Global Genes!
Attending the Global Genes Rare Corporate Alliance Meeting
At this meeting, we heard from Global Genes staff about initiatives that Global Genes is undertaking in the year ahead. CNF also presented on our Access to Critical Therapies initiative – a partnered initiative with Global Genes – that will be rolling out in 2020. More info to come in the new year!
Thank you to Global Genes for hosting this conference, and for the innovations you bring to your partnership with the Child Neurology Foundation and the entire rare disease community. It has been amazing to work together to act boldly on behalf of patients and families, and to map out work to enable better treatment and care for families on their journeys with rare disease.