Rare Disease Day 2023 Wrapped: What You Need to Take Away

Rob Long, Executive Director, Uplifting Athletes

Rob Long is a former All-American punter at Syracuse and has lived the rare disease journey. In December of 2010, late in his senior season, Rob was diagnosed with anaplastic astrocytoma, a rare and aggressive form of brain cancer. His prognosis at the time was less than encouraging, and his surgery, recovery and treatment took 16 months. Prior to his diagnosis, Rob was on a path to the NFL as a punter, but that opportunity was lost by the time he was healthy enough to train again. A graduate of Syracuse University, Rob pursued a masters in new media management from the S.I. Newhouse School of Public Communications. He also received a BS from the Martin J. Whitman School of Management.

Rob played football all four years for the Orange and was voted team captain by his teammates in his final two seasons. He became the second Executive Director of Uplifting Athletes at the end of 2018. Prior to taking over as the Executive Director, Rob served as Uplifting Athletes’ Director of Rare Disease Engagement for nearly two years. Rob has a steadfast commitment and connection to the rare disease community as a rare brain cancer survivor. As a former star college football student-athlete, his passion and drive to advance the mission of Uplifting Athletes is inspirational.

Richie Kahn, MPH, Co-Founder and Principal, Canary Advisors

Richie Kahn is a health policy professional by training, clinical researcher by trade and patient advocate by necessity. A rare disease patient and clinical trial participant himself, Richie is intensely passionate about better incorporating the patient perspective into the clinical development process. Through his work at Canary Advisors, he assists organizations set on patient-focused drug and device development by serving as an early-stage patient advocacy function; supporting regulatory patient advocacy; crafting patient-facing market access and reimbursement strategies; and providing clinical trial support services.

Jenn McNary, Co-Founder and Principal, Canary Advisors

Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate. Her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013 and the prestigious 2017 Meyer-Whalley instrument of change award. Formerly the director of outreach and advocacy at a Massachusetts based non-profit foundation, she was responsible for the organization of the largest US Food and Drug Administration(FDA) advisory committee hearing in history, with over 1,000 Duchenne advocates, families, clinicians and researchers in attendance. There are currently only three drugs approved for Duchenne, Exondys51, Vyondys53 and Emflaza; though in various roles, Jenn was involved in the approval process for all three. Jenn has unique experience in the drug development field, as a parent of children enrolled in the clinical trials, an advocate engaging with the regulators and as a patient engagement professional in the life sciences.

Currently, Jenn is the Co-Founder and Principal of Canary Advisors, a boutique patient advocacy consulting firm with a focus on regulatory and access patient engagement. Her other activities include serving as the Founder of One Rare, a non-profit formed to meet the needs of young adults with rare and chronic conditions.

Kyle Bryant, Director of rideATAXIA, Friedreich's Ataxia Research Alliance (FARA)

At age 17, Kyle and his family found out that his life would likely be cut short due to a progressively debilitating rare disease called Friedreich’s ataxia (FA).  In the face of this devastating news, riding his recumbent trike, Kyle has completed multiple cross country bike rides including an official finish of “The World’s Toughest Bike Race” – Race Across America, as part of 4-man Team FARA – this race is the focus of the award winning documentary “The Ataxian.”  In 2009 Kyle joined the staff of the Friedreich’s Ataxia Research Alliance (FARA) where he and his team built a nationwide series of bike rides called rideATAXIA which has raised over $11 million since 2007.  Kyle’s memoir, Shifting Into High Gear published in 2019. Kyle is co-host of the Two Disabled Dudes Podcast in which he and Sean Baumstark focus on living life beyond circumstances.

Christine Waggoner, President & Founder, Cure GM1 Foundation

Christine founded the Cure GM1 Foundation in April 2015 in honor of her daughter Iris and all those affected by GM1 gangliosidosis. Christine’s work at Cure GM1 has covered a broad range, including animal models, biomarkers, gene therapy, enzyme replacement therapy, patient registries, patient reported outcomes, newborn screening and the first-ever GM1 caregiver preferences study.

Christine received a B.A. from Brown University where she studied Visual Art and Computer Science. Christine holds 9 patents in computer graphics and has contributed to publications both pertaining to computer graphics and GM1 gangliosidosis. Christine is the 2023 Patient Advocacy Leadership Award issued by the WORLD Symposia. In 2017, Christine received the Sanofi TORCH award for outstanding patient advocacy.

(Host) Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials

Derek Ansel has worked in clinical research for more than 10 years with a focus almost exclusively in rare and pediatric diseases, including non-malignant hematology, autoimmune diseases, metabolic disorders, movement disorders and other genetic conditions. At Worldwide, he leads and supports corporate initiatives within rare and pediatric diseases and maintains relationships with over 60+ patient-focused advocacy organizations.

(Host) Amy Raymond, PhD, PMP, Senior Director, Therapeutic Strategy Lead, Rare Diseases, Worldwide Clinical Trials

Dr. Amy Raymond has been a drug discovery and development professional for 20+ years, including progressive roles in rare disease clinical operations and rare disease clinical strategy. Her clinical development experience spans all therapeutic areas and all stages of the clinical development lifecycle, from Natural History studies through in-patient Phase I trials, and Long-Term Follow Up and Post Marketing surveillance.