Behandling for Charcot-Marie-Tooth sykdom

The treatment for the neurological disease Charcot-Marie-Tooth (CMT) has for many years been symptomatic. No cure for this heredital neuropathy has been found. This raises some questions: Do the traditional symptomatic treatments really have effect on symptoms or prognosis? Are there any new and emerging treatments for the condition? These are the questions adressed in this assignment. Surgery as treatment was excluded from this assignment. A search of the relevant litterature revealed 20 articles on the topic. Some research has been done concerning traditional treatments such as physical training, treatment with footorthosis, and surgery for deformities. Interventional studies with physical training showed good effect in keeping up muscle strenght. The use of foot orthosis had no significant effect. Different pharmacological treatments been tested the last 25 years. The literature search revealed articles concerning treatment with Neuroptopin-3, creatine supplements, coenzyme Q10 and gangliosides. During the last decade, treatment with ascorbic acid (vitamin C), curcumin and progesterone antagonists have been studied. The most promising results are reported from the studies on Neurotropin-3, coenzyme Q10, curcumin and progesterone antagonists. New treatments for CMT, that aims to correct the phenotype, are RNA and gene based. These techniques have been a part of preclinical studies during the last few years. Transgenic rats and knock-out mice are good CMT-models that give great research opportunities. The results from these preclinical studies are so far very promising, and these techniques might in the future be used as treatment for the different mutations that cause CMT. Further research is necessary to find effective treatment for CMT, and there are multiple opportunities for future treatments of these patients