Duchenne Muscular Dystrophy treatment
November 20 2015 by
Ray Sahelian, M.D.

Duchenne muscular dystrophy is a frequent muscular disorder caused by mutations in the gene encoding dystrophin, a cytoskeletal protein that contributes to the stabilization of muscle fiber membrane during muscle activity. Affected individuals show progressive muscle wasting that generally causes death by age 30. Skeletal muscles become atrophied by muscular disorders such as muscular dystrophy, wasting and even aging. In addition to muscle atrophy, progressive muscle damage, inflammation and replacement of muscle fibers with fibrous and fatty tissues are observed in muscular dystrophy.
   Duchenne's muscular dystrophy is known to delay children's development of movement and coordination, but it may also slow language development. This severe inherited muscle disease affects 1 in 3500 boys worldwide.

Amino Acids. 2015. The L-arginine/NO pathway and homoarginine are altered in Duchenne muscular dystrophy and improved by glucocorticoids.

Alternative methods
CoQ10
Muscle Nerve. 2011. CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy. Cooperative International Neuromuscular Research Group Investigators. Children's National Medical Center, George Washington University, Washington, DC, USA. Corticosteroid treatment slows disease progression and is the standard of care for Duchenne muscular dystrophy (DMD). Coenzyme Q10 (CoQ10) is a potent antioxidant that may improve function in dystrophin-deficient muscle. We performed an open-label, "add-on" pilot study of CoQ10 in thirteen 5-10-year-old DMD patients on steroids. The primary outcome measure was the total quantitative muscle testing (QMT) score. Twelve of 16 children pcompleted the trial. Target serum levels of CoQ10 (≥2.5 μg/ml) were shown to be subject- and administration-dependent. Nine of 12 subjects showed an increase in total QMT score. Overall, CoQ10 treatment resulted in an 8.5% increase in muscle strength. Addition of CoQ10 to prednisone therapy in DMD patients resulted in an increase in muscle strength. These results warrant a larger, controlled trial of CoQ10 in DMD.

Biochim Biophys Acta. 1995. Two successful double-blind trials with coenzyme Q10 (vitamin Q10) on muscular dystrophies and neurogenic atrophies.Folkers K, Simonsen R.SourceInstitute for Biomedical Research, University of Texas at Austin, USA. Coenzyme Q10 (vitamin Q10) is biosynthesized in the human body and is functional in bioenergetics, anti-oxidation reactions, and in growth control, etc. It is indispensable to health and survival. The first double-blind trial was with twelve patients, ranging from 7-69 years of age, having diseases including the Duchenne, Becker, and the limb-girdle dystrophies, myotonic dystrophy. Charcot-Marie-Tooth disease, and the Welander disease. The control coenzyme Q10 (CoQ10) blood level was low and ranged from 0.5-0.84 microgram/ml. They were treated for three months with 100 mg daily of CoQ10 and a matching placebo. The second double-blind trial was similar with fifteen patients having the same categories of disease. Since cardiac disease is established to be associated with these muscle diseases, cardiac function was blindly monitored, and not one mistake was made in assigning CoQ10 and placebo to the patients in both trials. Definitely improved physical performance was recorded. In retrospect, a dosage of 100 mg was too low although effective and safe. Patients suffering from these muscle dystrophies and the like, should be treated with vitamin Q10 indefinitely.

I am writing on behalf of one of my relatives who his kid (6 years old) is suffering from Duchenne muscular dystrophy. He has tried to find a treatment and Coenzyme Q has been suggested, 1-2 per day. Actually I found your website through search on the internet. I would be so thankful if you could give some more information regarding which doze and how many tablet per day could be enough. And do you suggest this medicine for this disease?
   I do not have personal experience using coenzyme Q10 to know whether it is of benefit.

Creatine for muscular dystrophy
Creatine monohydrate as a therapeutic aid in muscular dystrophy.
Nutr Rev. 2006 Feb. Pearlman JP, Fielding RA. Nutrition, Exercise Physiology, and Sarcopenia Laboratory, Jean Mayer USDA Human Nutrition Research Center on Aging at Tufts University, Boston, Massachusetts, USA.
In recent years, dietary supplementation with creatine has been shown to enhance neuromuscular function in several diseases. Recent studies have suggested that creatine can be beneficial in patients with muscular dystrophy and other mitochondrial cytopathies, and may attenuate sarcopenia and facilitate rehabilitation of disuse atrophy. Though the mechanisms are still unknown, creatine has been shown to decrease cytoplasmic Ca2+ levels and increase intramuscular and cerebral phosphocreatine stores, providing potential musculoskeletal and neuroprotective effects.

Glutamine and amino acid supplement for Duchenne muscular dystrophy
Oral glutamine and amino acid supplementation inhibit whole-body protein degradation in children with Duchenne muscular dystrophy.
Am J Clin Nutr. 2006. Centre d'Investigation Clinique INSERM, Assistance Publique-Hopitaux de Paris, Hopital Robert Debre, Paris, France.
Glutamine has been shown to acutely decrease whole-body protein degradation in Duchenne muscular dystrophy (To improve nutritional support in Duchenne muscular dystrophy, we tested whether oral supplementation with glutamine for 10 d decreased whole-body protein degradation significantly more than did an isonitrogenous amino acid control mixture. Twenty-six boys with Duchenne muscular dystrophy were included in this randomized, double-blind parallel study; they received an oral supplement of either glutamine or an isonitrogenous, nonspecific amino acid mixture for 10 d. Oral glutamine or amino acid supplementation over 10 d equally inhibits whole-body protein degradation in DMD.

Green tea, EGCG, and Duchenne muscular dystrophy
Green tea extract and its major polyphenol epigallocatechin gallate improve muscle function in a mouse model for Duchenne muscular dystrophy.
Am J Physiol Cell Physiol. 2006. Laboratory of Pharmacology, Geneva-Lausanne School of Pharmaceutical Sciences, Univ. of Geneva, Switzerland.
In this study, the dystrophic mdx(5Cv) mouse model was used to investigate the effects of green tea extract, its major component (-)-epigallocatechin gallate, and pentoxifylline on dystrophic muscle quality and function. Three-week-old mdx(5Cv) mice were fed for either 1 or 5 wk a control chow or a chow containing the test substances. Histological examination showed a delay in necrosis of the extensor digitorum longus muscle in treated mice. Mechanical properties of triceps surae muscles were recorded while the mice were under deep anesthesia. Phasic and tetanic tensions of treated mice were increased, reaching values close to those of normal mice. The phasic-to-tetanic tension ratio was corrected. Finally, muscles from treated mice exhibited 30-50% more residual force in a fatigue assay. These results demonstrate that diet supplementation of dystrophic mdx(5Cv) mice with green tea extract or EGCG protected muscle against the first massive wave of necrosis and stimulated muscle adaptation toward a stronger and more resistant phenotype.

Idebenone
Lancet. 2015. Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial. Cardiorespiratory failure is the leading cause of death in Duchenne muscular dystrophy. Based on preclinical and phase 2 evidence, we assessed the efficacy and safety of idebenone in young patients with Duchenne muscular dystrophy who were not taking concomitant glucocorticoids. :In a multicentre phase 3 trial in Belgium, Germany, the Netherlands, Switzerland, France, Sweden, Austria, Italy, Spain, and the USA, patients (age 10-18 years old) with Duchenne muscular dystrophy were randomly assigned in a one-to-one ratio with a central interactive web response system with a permuted block design with four patients per block to receive idebenone (300 mg three times a day) or matching placebo orally for 52 weeks. Idebenone reduced the loss of respiratory function and represents a new treatment option for patients with Duchenne muscular dystrophy.

Research
Mediators Inflamm. 2013. Cytokines and chemokines as regulators of skeletal muscle inflammation: presenting the case of Duchenne muscular dystrophy. Infiltration of skeletal muscle by inflammatory cells is an important facet of disease pathophysiology and is strongly associated with disease severity in the individual patient. In the chronic inflammation that characterizes Duchenne muscle, cytokines and chemokines are considered essential activators and recruiters of inflammatory cells. In addition, they provide potential beneficiary effects on muscle fiber damage control and tissue regeneration. In this review, current knowledge of cytokine and chemokine expression in Duchenne muscular dystrophy and its relevant animal disease models is listed, and implications for future therapeutic avenues are discussed.

Emails
Would like to know if vitamin B17 is beneficial for treatment of Duchenne Muscular Dystrophy.
   A. I do not have experience in the use of this vitamin for this condition.