Challenges and strategies for clinical trials in propionic and methylmalonic acidemias
Jerry Vockley, Barbara K. Burton, Agnieszka Jurecka, Jitendra Ganju, Beth Leiro, Roberto T. Zori, Nicola Longo
Topics & Concepts
Methylmalonic acidemiaClinical trialMedicineDiseaseIntensive care medicineClinical study designNatural history studySelection (genetic algorithm)Natural historyPediatricsInternal medicineComputer scienceArtificial intelligenceMetabolism and Genetic DisordersGenomics and Rare DiseasesFolate and B Vitamins Research