Evidence supporting the use of: Human growth hormone
For the health condition: Muscular Dystrophy

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Synopsis

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

Human growth hormone (hGH) has been studied as a therapeutic option for individuals with muscular dystrophy (MD), particularly Duchenne Muscular Dystrophy (DMD). The rationale is based on hGH’s anabolic effects, which promote protein synthesis and muscle growth. Some early studies and case series suggested that hGH might improve muscle mass, growth velocity, and perhaps even muscle strength in children with DMD, especially when combined with physical therapy or corticosteroids. However, the evidence base is limited and mixed.

Several clinical trials have shown modest increases in lean body mass but have generally failed to demonstrate significant improvements in muscle function or long-term outcomes. Concerns about potential side effects—including increased risk of glucose intolerance, pseudotumor cerebri, and exacerbation of cardiomyopathy—have tempered enthusiasm for routine use. As a result, major clinical guidelines do not currently recommend hGH as a standard therapy for MD, and its use is considered experimental or adjunctive.

In summary, while there is some scientific rationale and limited clinical evidence for the use of hGH in muscular dystrophy, the overall quality and quantity of evidence are low, and the risk-benefit ratio remains uncertain. Larger, well-controlled studies are needed before hGH can be recommended as a standard treatment for MD.

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