Glucocorticosteroid Diseases Biological marker Mecp2 MND Errance diagnotique Amyotrophic Lateral Sclerosis Coagulation factor IX CRISPR/SaCas9 Longitudinal progression Adult patients Disease modifiers Dicer ALS Epigenetic changes Adult SMA IRM FTD Cellules souches musculaires Effector T cells Antisense oligonucleotides AAV Brain damage Bioinformatics Bone involvement Extremely preterm infants Adenosine Cartilage and bone regeneration Maladie neuromusculaire Chondrocytes Brain injury Clinical trial Mouse model Microglia Intra-CSF delivery Aav10 ASO ASOs IPSCs Early-onset sepsis Clinical trials Albumin gene targeting Bone development Amyotrophie spinale Icv Fetal growth restriction Brain Disease heterogeneity Brain imaging Long-term handicap Duchenne Muscular Dystrophy Modèle murin AICD Biomarker Genetical therapy Biomarkers C9orf72 FOXO3a Prematurity Intra-uterine growth restriction MRNP assembly Neuromuscular disease Genetics Gene transfer Calcium handling ERK1/2 signaling Clinical markers SMN Blood brain barrier Melatonin DPRs G-Secretase DTI IUGR MiRNA MUNIX Maternal behavior MRI FGR Lentiviral vectors Brain development Brain MRI Les paramètres respiratoires Spinal muscular atrophy GeneRide 3xTgAD Mice Dilated cardiomyopathy Distal myopathy CNS Cell reprogramming Cell stemness GABA Inflammation Fabry disease lysosomal storage disorders adeno asociated virus-9 LMNA Functional outcomes Maternal malnutrition Skeletal muscle Gene therapy Cofilin-1