Symposium on Best Practices in Clinical Study Design for Rare Diseases

April 29-30 in Washington, D.C.


Recently I had the privilege of attending and participating in the Symposium on Best Practices in Clinical Study Design of Rare Diseases in Washington, D.C. The symposium was organized in collaboration with Children’s National Medical Center (CNMC), Clinical and Translational Science Institute of Children’s National (CTSI), The George Washington University, and Research in Pediatric Developmental Pharmacology Centers (RPDP).  This symposium was focused on setting the stage for discussing rare diseases drug development and clinical trials and challenges in the landscape focusing on Cystic Fibrosis and Duchenne muscular dystrophy.


I took extensive notes which are linked to below. I hope you find these helpful!


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