Climbing the Advocacy Summit

By Sara Casagrand

Last week, members of the SmithSolve team had the opportunity to attend the largest-yet Global Genes RARE Patient Advocacy Summit in Irvine, CA. It was encouraging to see the growth and excitement surrounding this important learning and networking event in rare disease.

The conference kicked off with an inspiring story from Simon Wheatcroft, an ultra-marathon runner who lost his sight at 17 years old from retinitis pigmentosa. The founder of &Adapt, Wheatcroft shared his story of re-learning how to run, despite his blindness. He finished a 260-mile journey from Boston to New York and topped it off by completing the New York City Marathon.

Throughout the week, there were engaging, informative and collaborative sessions on all aspects of rare disease such as emerging therapies, newborn screening, advocacy, and the critical role patients play in research and drug development. For the first time, the conference included a RARE Investor and Partnering Forum to discuss models for building and funding rare disease companies. Panelists describe the many ways biopharma companies large and small are collaborating with scientists, academic centers and patient advocates to advance treatments and secure funding.

During the RARE Champion of Hope Awards, a long-time patient advocate and our friend, Barbara Wuebbels, VP of Global Patient Relations at Audentes Therapeutics, Inc. was awarded the RARE Champion in Advocacy award, a truly fitting and well-deserved honor. We congratulate all the honorees for their hard work and dedication to the rare disease community.

We also walked the blue carpet with our colleagues at the Sixth Annual Tribute to Champions of Hope Gala and heard moving performances from Sparsh Shah, Andrew McMahon in the Wilderness, and more. The evening was a celebration of the progress that’s been made, but also a reminder of the work yet to come.

Congratulations to the Global Genes team for all of the planning, passion and hard work they put into making the RARE Patient Advocacy Summit a success!