In parallel, rare disease and (tailored) genetic therapies are gaining mainstream attention with the Food and Drug Administration (FDA) approval of the antisense oligonucleotide (Nusinersen) as well as the gene therapy (onasemnogene abeparvovec) for Spinal muscular atrophy (SMA) [34, 35] and the first personalized antisense oligonucleotide (Milasen) for CLN7-related neurodegeneration [36]. Here, MFSD8 is linked to proximal spinal muscular atrophy.