Principles for interactions with biopharmaceutical companies: The development of guidelines for patient advocacy organizations in the field of rare diseases
Stein, S., Bogard, E., Boice, N., Fernandez, V., Field, T., Gilstrap, A., Kahn, S. R., Larkindale, J., & Mathieson, T. (2018). Orphanet Journal of Rare Diseases, 13(1), 18. https://doi.org/10.1186/s13023-018-0761-2
Research
RARE-X: A patient-driven approach for collecting symptom and patient-reported outcome data in rare diseases
Vogel-Farley, V., Trzupek, K., Gosar, J., Hobbs, K., Wentworth, K., Michaels, B., Beek, G., Dang, T., Abramson, M., O’Boyle, M., Boice, N., Son Rigby, C., & Talebizadeh, Z. (2025). Genetics in Medicine, 28(2), 101634. https://doi.org/10.1016/j.gim.2025.101634
Breaking barriers in rare disease research: The RARE-X Open Science Data Challenge as a model for collaborative innovation and community partnership
Trzupek, K., Bhargava, R., Kuan, C., Sie, F., Vogel-Farley, V., Hobbs, K., Chung, V., Diaz, M., Son-Rigby, C., Geraci, J., & Albrecht, J. (2025). HGG Advances, 6(3), 100462. https://doi.org/10.1016/j.xhgg.2025.100462
Dental complications in homocystinurias
Chapman, K. A., Bartke, D., Vogel-Farley, V., Cobb, M., & Chapman, M. (2023). Molecular Genetics and Metabolism Reports, 36, 100999. https://doi.org/10.1016/j.ymgmr.2023.100999
Principles for interactions with biopharmaceutical companies: The development of guidelines for patient advocacy organizations in the field of rare diseases
Stein, S., Bogard, E., Boice, N., Fernandez, V., Field, T., Gilstrap, A., Kahn, S. R., Larkindale, J., & Mathieson, T. (2018). Orphanet Journal of Rare Diseases, 13(1), 18. https://doi.org/10.1186/s13023-018-0761-2
