PROGRESS FSHD Remote assessment and artificial intelligence to validate new measures, biomarkers, and therapeutic targets for facioscapulohumeral muscular dystrophy (FSHD) Rare diseasesDatabases
RASores Preclinical approaches for the treatment of RASopathy patients through a multi-omic study of their pathophysiology using a deeply phenotyped cohort in a dedicated European registry Rare diseasesDatabasesGenetics/Heredity