RYR-1-Related Diseases International Research Workshops
2025 RYR-1-Related Diseases Patient-Led International Research Workshop: Novel Perspectives, Treatments & Interventions
July 23 – July 24, 2025
Hyatt Regency Pittsburgh International Airport
Pittsburgh, PA, USA
The RYR-1 Foundation will be hosting its second RYR-1-RD Patient-Led International Research Workshop at the Hyatt Regency Pittsburgh International Airport. The Research Workshop will immediately precede The RYR-1 Foundation International Family Conference 2025.
The Research Workshop will bring together an international group of experts, researchers, medical professionals, trainees, and affected families focused on RYR-1-Related Diseases (RYR-1-RD) to share knowledge and exchange ideas.
Planning the Research Workshop are the Co-Directors, composed of members of The RYR-1 Foundation’s Board of Directors, Board of Advisors, and the Scientific Advisory Board. The Co-Directors are: Michael F. Goldberg, MD, MPH; Johanna Lanner, PhD; Livija Medne, MS, LCGC; Payam Mohassel, MD; Jinson Paul, MBA; Emily Pedersen; and Mairéad Ryan, BS, V27.
Testimonials from patients and families from the RYR-1-RD Community, a key part of the Research Workshop, will be featured in each session. The agenda sessions are: Diagnosis & Genetics of RYR-1-RD; Clinical Trial Readiness; Novel Insight into RyR1 Interplay and Cell Pathophysiology; RyR1 and Knowledge Gained from Animal Models; Therapeutic Pipeline; and Physical Therapy & Exercise Approaches in RYR-1-RD. The Research Workshop agenda can be viewed below.
This Research Workshop is by invitation only and a limited number of people can be accommodated. A manuscript will be produced from the 2025 Research Workshop and will be shared publicly following the Research Workshop.
2025 RYR-1-Related Diseases Patient-Led International Research Workshop: Novel Perspectives, Treatments & Interventions Code of Conduct
The Research Workshop Co-Directors are dedicated to providing a harassment-free Research Workshop experience for everyone, regardless of gender, gender identity and expression, sexual orientation, disability, physical appearance, body size, race, age, or religion. This policy is in place to ensure the unhindered exchange of scientific ideas and critiques, which are critical to all science conferences/workshops. We will not tolerate harassment of participants in any form. Our Research Workshop material includes information on how to report code of conduct violations to staff of The RYR-1 Foundation. Participants violating these rules will be expelled from the Research Workshop. The Research Workshop Code of Conduct will be strictly enforced. We have a zero-tolerance policy for discriminatory behavior based on identity, as well as physical, verbal, or sexual harassment. The code of conduct will be posted on the Research Workshop website and attendees will be asked to read and acknowledge the code of conduct prior to attending.
For more information on the Research Workshop, contact Lena Leghart.
Become A Sponsor and Make A Difference Together!
Through the Patient-Led International Research Workshop Sponsorship Program, The RYR-1 Foundation offers an opportunity to organizations with shared interests to partner with us. As a sponsor of the Patient-Led International Research Workshop, your organization directly supports the work of The RYR-1 Foundation in finding a cure for RYR-1-Related Diseases (RYR-1-RD). Your organization also receives benefits that help you meet your goals, including joining a global community.
For more information on Sponsorship Opportunities, contact Patricia Raffaele.
2025 Research Workshop Sponsorship Opportunities
2025 Sponsor Recognition
The RYR-1 Foundation extends its heartfelt thanks to Eagle Pharmaceuticals for their Platinum Sponsorship of the 2025 RYR-1-Related Diseases Patient-Led International Research Workshop: Novel Perspectives, Treatments & Interventions. We are truly grateful for Eagle’s sponsorship, which is instrumental in making this unique workshop possible. On behalf of all individuals and families affected by RYR-1-RD, we sincerely thank Eagle Pharmaceuticals for their invaluable partnership.
2025 Grant Recognition
The RYR-1 Foundation extends its heartfelt thanks to the Muscular Dystrophy Association (MDA) for their generous support of the 2025 RYR-1-Related Diseases Patient-Led International Research Workshop: Novel Perspectives, Treatments & Interventions. We are deeply grateful for MDA’s grant, which plays a pivotal role in making this unique workshop possible. On behalf of all individuals and families affected by RYR-1-RD, we offer our sincerest gratitude to MDA for their partnership.
2022 RYR-1-Related Diseases International Research Workshop: From Mechanisms to Treatments
July 21 – July 22, 2022
Hyatt Regency Pittsburgh International Airport
Pittsburgh, PA, USA
This was the first-ever patient-led international research workshop devoted exclusively to RYR-1-Related Diseases (RYR-1-RD), preceding the 2022 RYR-1 International Family Conference. With 45 in-person attendees and ten virtual attendees from 11 different countries, this workshop provided a much-needed forum for the leading international RYR-1-RD experts and a group of affected individuals to share knowledge, exchange ideas, form collaborations, and develop new strategies for finding therapies. The Workshop Co-Directors included a diverse group of researchers/clinicians in RYR-1-RD, members of The RYR-1 Foundation’s Scientific Advisory Board (SAB), and RYR-1-RD community representatives.
2022 Research Workshop One-Page Flyer
2022 Research Workshop Program
The RYR-1 Foundation is elated to report that the manuscript arising from this workshop has undergone editorial review and has now been published in the Journal of Neuromuscular Diseases, the leading medical journal for neuromuscular disease. This important publication will serve to educate a worldwide audience on the current state of RYR-1-RD research, and clinical care. As important, this document will also serve to guide future research efforts to expedite the development of effective therapies.
2022 Research Workshop Publication
2022 Grant Recognition
The RYR-1 Foundation would like to thank the Muscular Dystrophy Association (MDA) and the National Institutes of Health (NIH) for their support of the 2022 RYR-1-Related Diseases International Research Workshop: From Mechanisms to Treatments. We are extremely grateful for the MDA’s and NIH’s grants to help us host this one-of-a-kind workshop. On behalf of all individuals and families affected by RYR-1-RD, The RYR-1 Foundation expresses our sincerest gratitude.
Funding for this conference was made possible (in part) by (1R13AR081619-01) from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
2022 RYR-1-Related Diseases International Research Workshop Gallery
