A mutation in one copy of the gene PMP-22 (Peripheral myelin protein 22, 17p11.2) that makes the peripheral myelin protein causes haploinsufficiency, where the activity of the normal gene is insufficient to compensate for the loss of function of the other gene.
Medwell Capital obtained an exclusive worldwide license to proprietary technology developed at the MS Patient Care and Research Clinic at the University of Alberta for the treatment of MS. The compound, dirucotide (MBP8298), is a synthetic myelin basic protein peptide composed of 17 amino acids which is intravenously injected (every six months) into MS patients as a therapeutic treatment.