Collaborating with advocacy partners for gene therapy research
Patient advocacy organizations are critical for advancing gene therapy research and clinical trial success. Throughout our history, we have established significant, meaningful relationships with advocacy groups centered on patients and the urgent need many genetic diseases create.
Advocacy groups are some of our greatest collaborators. Our close associations are key to prioritizing clinical candidates, establishing research protocols, and advancing technology innovation.
Our advocacy partners
We partner with advocacy groups to support research, education and events that benefit patients, advance science and improve outcomes.
- Muscular Dystrophy Association
- Parent Project Muscular Dystrophy
- CureDuchenne
- AMDA – Acid Maltase Deficiency Association
- International Pompe Association
- Canadian Association of Pompe
- New Zealand Pompe Network
- Pompe Support Network
- Speak Foundation
- CureLGMD2i Foundation
- Angelman Syndrome Foundation
- Defeat MSA Alliance
- Nationwide Children’s Hospital
- North Carolina Biotechnology Center
- North Carolina Board of Science & Technology
The RhiaPharmaceutical pipeline
Our pipeline is the direct result of listening to patients, their families, their care teams, and patient advocacy organizations.
