These medications include also newly FDA and/or EMA approved drugs, such as enzymatic replacement therapy (ERT) for Pompe disease, antisense oligonucleotides (ASO) for Duchenne Muscular Dystrophy (DMD) patients with deletions amenable to exon 51 skipping, splicing-modulator ASO and gene therapy for Spinal Muscular Atrophy (SMA), and messenger-RNA interfering molecules for transthyretin (TTR) amyloidotic neuropathy. This evidence concerns the gene TTR and spinal muscular atrophy.