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. Author manuscript; available in PMC: 2022 Mar 1.
Published in final edited form as: Trends Cell Biol. 2020 Dec 1;31(3):197–210. doi: 10.1016/j.tcb.2020.11.003

Figure 4: Potential treatments for FKRP-associated dystroglycanopathies.

Figure 4:

Preclinical studies aiming to develop treatments for fukutin related protein (FKRP)-related disorders include small molecules, gene and cell therapy. Small molecules, like ribitol, nicotinamide adenine dinucleotide (NAD+), 4-(4-bromophenyl)-6-ethylsulfanyl-2-oxo-3,4-dihydro-1H-pyridine-5-carbonitrile (4BBNit), pentetic acid, prednisolone combine with alendronate and the use of tamoxifen have shown the ability to improve disease phenotype. Gene therapy studies with FKRP, like-acetylglucosaminyltransferase (LARGE1) and Beta-1,4 N-acetylgalactosaminyltransferase 2 (B4GALNT2) have shown promising results. Cell therapy studies using engineered satellite cells and the tranplantation of pluripotent stem cell (PSC)-derived myogenic progenitors have also susccesfully rescued the FKRP pathology.