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About the Journal
Translational Science of Rare Diseases features independent, original research articles as well as in-depth reviews of specific rare diseases and therapeutics and opinion pieces from regulators, patient advocates and leading industry and academic researchers focused on rare diseases.
The journal has forged strong connections with and access points to the global rare diseases network that transcend national boundaries and stimulate multidisciplinary and international collaboration.
“Taking the broadest perspective, the journal is intended to engage the energies of the international medical community and encompass fundamental, translational, and clinical trial design to advance studies related to rare diseases. Our goal has been and remains the creation of a platform that creates conversations leading to effective treatment and, ultimately, a cure.”
--Editors-in-Chief, Rashmi Gopal-Srivastava, PhD, and Alan K. Percy, MD
- The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study
- Transition of care of patients with eosinophilic gastrointestinal diseases: Challenges and opportunities
- Efficacy and safety of long-term sirolimus use as part of multidisciplinary care in a pediatric patient with CLOVES syndrome: Case report
- Report of the 2021 Primary Ciliary Dyskinesia Foundation annual meeting