A vitamin could help treat Duchenne muscular dystrophy

Date:

October 19, 2016

Source:

Ecole Polytechnique Fédérale de Lausanne

Summary:

Researchers are working on a new strategy to combat one of the most severe forms of muscular dystrophy. Rather than acting on the defective gene, they are using large doses of a vitamin.

Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general paralysis, and most patients die of respiratory failure. The disease is caused by a genetic mutation that prevents a protein required to keep muscle cells intact from being produced. While most research focuses on repairing the defective gene, researchers at EPFL have come up with a different strategy. As part of their work on nutrition and aging, they discovered that large doses of a vitamin called nicotinamide riboside were remarkably effective in countering the progress of the disease in animals. Their work has been published in Science Translational Medicine.

Journal Reference:

1. D. Ryu, H. Zhang, E. R. Ropelle, V. Sorrentino, D. A. G. Mazala, L. Mouchiroud, P. L. Marshall, M. D. Campbell, A. S. Ali, G. M. Knowels, S. Bellemin, S. R. Iyer, X. Wang, K. Gariani, A. A. Sauve, C. Canto, K. E. Conley, L. Walter, R. M. Lovering, E. R. Chin, B. J. Jasmin, D. J. Marcinek, K. J. Menzies, J. Auwerx. NAD repletion improves muscle function in muscular dystrophy and counters global PARylation. Science Translational Medicine, 2016; 8 (361): 361ra139 DOI: 10.1126/scitranslmed.aaf5504