Synopsis

Source of validity: Scientific
Rating (out of 5): 2

Phosphocreatine (often supplemented as creatine monohydrate) has been investigated as a supportive therapy for muscular dystrophy, particularly Duchenne Muscular Dystrophy (DMD). The rationale stems from the role of phosphocreatine in rapid ATP regeneration in muscle cells, which is compromised in muscular dystrophies due to progressive muscle wasting and energy deficits.

Several small clinical studies and pilot trials have explored creatine supplementation in muscular dystrophy patients. Some evidence suggests modest improvements in muscle strength and function, as well as increased fat-free mass in children and adults with DMD and other neuromuscular disorders. For example, a randomized, double-blind, placebo-controlled trial published in The Lancet (2004) indicated that creatine supplementation improved strength in boys with DMD. However, the effect sizes were generally small, and not all studies found significant benefits. Additionally, these studies were typically short-term and involved small sample sizes.

Systematic reviews and meta-analyses (e.g., Kley et al., 2013, Neurology) conclude that while creatine is well-tolerated and may provide minor functional improvements, the evidence base is limited and does not establish creatine/phosphocreatine as a standard treatment. No large-scale, long-term trials have proven substantial clinical benefit, and creatine is considered an adjunct rather than a primary therapy.

In summary, there is some scientific support for the use of phosphocreatine (as creatine supplementation) in muscular dystrophy, but the evidence is limited and effects are modest, justifying a rating of 2/5.

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