Miopatie mitocondriali primarie: i ricercatori esplorano nuove vie per il trattamento
Recent insights into the nature of mitochondrial diseases have led to novel approaches in treating them. Mitochondrial diseases are “genetic,” which means that patients are born with inherited errors in their DNA that reduces the performance of their mitochondria. Mitochondria are found within cells and are responsible for producing energy for the cells. In patients with mitochondrial disease cells that require a lot of energy, such as the skeletal muscles needed for walking, don’t produce enough energy to function as they should.
Le attuali opzioni di trattamento per le malattie mitocondriali rimangono limitate a fornire sollievo sintomatico, piuttosto che colpire direttamente la causa di quei sintomi e della malattia: la mancanza di mitocondri sani e funzionanti .
One of the areas that may directly improve mitochondrial performance is increasing the activity of a protein called peroxisome proliferator-activated receptor (PPAR receptor delta). PPARs are found in muscle and other cells and activate the creation of mitochondria. Earlier studies of investigational medications that specifically worked by activating the PPAR delta receptors in respiratory muscle have shown it stimulated the mitochondria and improved muscles’ ability to function. It may even be that stimulating the PPAR delta can increase mitochondrial function enough to improve energy production and affect disease.
Per le persone con miopatie mitocondriali primarie (PMM), è rassicurante sapere che nuovi studi continuano ad esplorare gli attivatori di PPAR delta come un possibile modo per stimolare l'attività mitocondriale, aumentare la produzione di energia e migliorare le prestazioni delle cellule muscolari scheletriche.
Fonte: Hassani A, et al. Miopatie mitocondriali: sviluppi nel trattamento. Opinione corrente in Neurologia 2010, 23:459-465
KR BLOG-PMM-1003 v3
