N-Acetyl L-Leucine Nicotinamide Riboside (NAD+) Triheptanoin Intra-Erythrocyte Dexamethasone Hematopoietic stem cell transplant & FiCAT Nanoparticle chemistry with gene augmentation Transposons method T-Car Splice site mutations Intrabio, UK Oslo University Hospital, Norway Queensland Children’s Hospital Brisbane, Australia Erydel, Italy CUN (Navarra) & UPF (Barcelona), Spai University of Nottingham, UK University of Granada, Spain Sheba Medical Centre, Israel Harvard and Boston Children’s Hospital, USA