Complex Feasibility Assessment in Low-to- Middle Income Countries
Complex Feasibility Assessment in Low-to- Middle Income Countries
3 PagesCASE STUDY Complex Feasibility Assessment in Low-to- Middle Income CountriesComplex Feasibility Assessment in Low-to-Middle Income Countries Service Area Early Engagement STUDY DESCRIPTION Determining the feasibility of adeno-associated virus (AAV) mediated gene transfer (AAV2/8-FIX Padua) for severe hemophilia B in adults and post-pubertal adolescents in low-to-middle income countries (LMICs) Despite exponential growth in gene therapy product development and testing in clinical trials, few patients in LMICs have access to novel and investigational treatments. Hemophilia patients in most LMICs lack access to even the standard care available in developed countries. A large research hospital approached PRA to assess the feasibility of an AAV-mediated gene transfer for severe hemophi
