Prednisone wasn't effective when tested on eight people with facioscapulohumeral dystrophy. Prednisone and related drugs are routinely used in myasthenia gravis, Lambert-Eaton myasthenic syndrome, polymyositis and dermatomyositis. McArdle's disease looks promising in experiments in sheep, and human trials may not be far off. That's the word from MDA grantees John Howell of the Division of Veterinary and Biomedical Sciences of Murdoch University in Perth, Western Australia, and George Karpati of the Department of Neuromuscular Research at Montreal Neurological Institute. They therefore have limited ability to exercise and can severely damage their muscles if they overuse them.
Kidney damage can occur as a result of the muscle breakdown.
Inserting a corrected phosphorylase gene would theoretically improve their exercise capacity and protect their muscles and kidneys. Results will be reported to your doctor. There is only medicine that has been adequately tested and medicine that has not, medicine that works and medicine that may or may not work. What matters is the testing process. Angell and Kassirer decry the 1994 passage of the Dietary Supplement Health and Education Act, which made substances considered dietary supplements nearly exempt from Food and Drug Administration regulation.