Myopathies mitochondriales primaires : les chercheurs explorent de nouvelles voies thérapeutiques
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.
Les options thérapeutiques actuelles pour les maladies mitochondriales se limitent à apporter un soulagement symptomatique, plutôt que d'agir directement sur la cause de ces symptômes et de la maladie : l'absence de mitochondries saines et fonctionnelles.
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.
Pour les personnes atteintes de myopathies mitochondriales primaires (PMM), il est rassurant de savoir que de nouvelles études continuent d'explorer les activateurs du PPAR delta comme moyen possible de stimuler l'activité mitochondriale, d'augmenter la production d'énergie et d'améliorer les performances des cellules musculaires squelettiques.
Source : Hassani A, et al. Mitochondrial Myopathies : developments in treatment. Opinion courante en neurologie 2010, 23:459-465
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