Conference provides insights for rare disease organizations

The annual Global Genes RARE Patient Advocacy Summit held last month in San Diego is one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, and partners. Sessions provided insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies to take home.

Chief Scientific Officer Eric Scheeff attended on behalf of IDefine and came away optimistic and energized.

“It’s exciting just how much is happening in this space, how it’s exploding with activity, and how there are so many people focused on rare diseases,” said Scheeff. “So many companies are springing up because they see an opportunity here. That’s incredibly exciting because it means as an organization, we have options about who we work with and what we explore preclinically in terms of a possible treatment.”

As a scientist, Scheeff attended primarily technical sessions.

“One of the most important ones to me was on how to grow your organization and decide how to deploy precious research dollars, because that’s my focus.”

In addition to formal sessions, informal conversations also proved fruitful.

“The attendees are like this family of people who all represent different disorders, and we’re facing a lot of the same challenges,” said Scheeff. “A lot of the best conversations I had were just talking to people about what they’re up to. They would tell us they’re doing XYZ and that would be new information, opening new possibilities for IDefine. So we were learning from each other.

“We’re stronger together as a KS group. We’re also stronger together as a whole rare disease community working together. Those connections are just so critical, and that’s why Global Genes exists.”

Other Key Takeaways for IDefine

Scheeff’s key takeaways from the conference include that the Kleefstra Syndrome community is in the same boat as other rare diseases.

“I think it is helpful to understand that a lot of the difficulties we’re facing as community are being faced by everybody else. We shouldn’t feel alone. That goes to both as an organization and as a community of individuals. Almost everybody there is also the parent of a special needs kid.”

Scheeff also thinks it’s important to remember that being a rare disease parent and growing a rare disease organization are incredibly challenging.

“No one has all the answers. The people who did presentations don’t have all the answers. The people in the community don’t have all the answers. Everybody is figuring it out as they go.  It’s sort of reassuring that we don’t have to have all the answers to take steps forward as an organization and as a community.”

The Global Genes conference includes tracks for adults with rare diseases, care givers, and skill building for organizations. Next year’s conference will again be held in September in San Diego.