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These are systematic reviews of primary research. They explore the evidence for and against the effectiveness and appropriateness of treatments (medications, surgery, education, etc).

We provide a link to each plain language summary on the Cochrane web site, which are often available in English and several other languages. From there you can link to the full report and download a PDF (not available in all countries).


The Cochrane Collaboration, 2014

This is the best source of information about whether or not any particular pharmacological or nutritional treatment is effective. The review was prepared by Dr Ros Quinlivan (UK), Dr Andrea Martinuzzi (Italy) and Dr Benedikt Schoser (Germany). The review's conclusions for clinical practice are: “Although there was low quality evidence of improvement in some parameters with creatine, oral sucrose, ramipril and a carbohydrate-rich diet, none was sufficiently strong to indicate significant clinical benefit.”


The Cochrane Collaboration, 2011

The objective of the review was to systematically assess the evidence for physical training to improve exercise capacity and function in daily life in people who have McArdle Disease. The authors are Dr Ros Quinlivan (London, UK), Dr John Vissing (Denmark), Dr David Hilton-Jones (Oxford, UK), and Dr John Buckley (Chester, UK). Whilst anecdotally we know that in clinical situations McArdle people can improve greatly through regular aerobic exercise, the review found that there was not yet sufficiently robust scientific evidence. 

The key finding was that further studies would be safe to conduct:

“Implications for practice - The results of small numbers of subjects in three non-randomlsed studies suggest that aerobic training is safe for people with McArdle disease; however, there is no evidence from randomised controlled trials for therapeutic benefit of aerobic exercise.

Implications for research - The proof of principle studies described in the discussion of this review suggest that it would be worthwhile to undertake randomised controlled trials of aerobic training and that such studies would be safe to perform.”

Physical training

The Cochrane Collaboration, 2013

Hereditary muscle diseases usually lead to a progressive muscle weakness. Treatment is mainly symptomatic because curative therapies are lacking. Creatine, a popular nutritional supplement among athletes, improves muscle performance in healthy individuals. This is an update of our review evaluating creatine treatment in muscle disorders that was first published in 2007. At this update we identified no new studies but we had previously found 14 randomised controlled trials with 364 participants which met our defined selection criteria. The methodological quality of these studies was high, with only one exception. Analysis of pooled results showed a significant increase in muscle strength in muscular dystrophies and an improvement in activities of daily living in muscular dystrophies and inflammatory myopathies during creatine treatment compared to placebo. Significant adverse events occurred only in people with glycogen storage disease type V presenting as an increase in muscle pain episodes and impairment in activities of daily living. 

The key finding in regard to McArdle disease was disappointing:

“High-dose creatine treatment impaired activities of daily living and increased muscle pain in McArdle disease.

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