Fig. 3.

a Neuroprotection via small molecules. PEG-IGF1 is a neurotrophic factor. Mab2256 is a monoclonal antibody with an agonistic effect on tyrosine kinase receptor C that has neuroprotective effect. b Enhancement of muscle functionality. Small molecules to enhance the muscle contractility (CK2127107) and mass of the muscles (myostatin activation inhibitor) can be considered. c mRNA modulation. ASO and small molecules can modulate mRNA splicing and transcription increasing the IGHMBP2 protein level. d Cell transplantation. Human induced pluripotent stem cells (iPSCs) can be generated from adult fibroblasts. The iPSCs are then differentiated into neural stem cells (NSCs) or GFP-positive MNs and transplanted into a SMARD1 mouse to improve the phenotype of the animal. e Gene therapy. This approach is based on the replacement of the defective gene using self-complementary adeno-associated viral vectors