Enzyme replacement therapy: current challenges and drug delivery prospects via extracellular vesicles
Amanda Silva, Corinne Sagné, Florence Gazeau, Ibane Abasolo
Abstract
Rare Disease and Orphan Drugs Journal (RDODJ) is an international, peer-reviewed, open access journal. RDODJ will report on scientific advances in the genetics of rare diseases, the molecular basis of the pathologies, and translational research on diagnosis, prevention and treatment. In addition, RDODJ aims to provide a forum for scientific studies and discussion covering the important regulatory, socio-economic and human science issues related to rare diseases and orphan drugs.
Topics & Concepts
Orphan drugEnzyme replacement therapyExtracellular vesiclesRare diseaseMedicineTranslational researchDiseaseDrug discoveryEngineering ethicsPharmacologyIntensive care medicineBioinformaticsBiologyPathologyEngineeringCell biologyExtracellular vesicles in disease