Treatment
There is no current cure for DMD, although phase 1-2a trials with exon-skipping treatment for certain mutations have halted decline and produced small clinical improvements in walking.
Treatment is generally aimed at controlling the onset of symptoms to maximize the quality of life, and include the following:
- Corticosteroids such as prednisolone and deflazacort increase energy and strength and defer severity of some symptoms.
- Randomised control trials have shown that beta2-agonists increase muscle strength but do not modify disease progression. Follow-up time for most RCTs on beta2-agonists is only around 12 months and hence results cannot be extrapolated beyond that time frame.
- Mild, non-jarring physical activity such as swimming is encouraged. Inactivity (such as bed rest) can worsen the muscle disease.
- Physical therapy is helpful to maintain muscle strength, flexibility, and function.
- Orthopedic appliances (such as braces and wheelchairs) may improve mobility and the ability for self-care. Form-fitting removable leg braces that hold the ankle in place during sleep can defer the onset of contractures.
- Appropriate respiratory support as the disease progresses is important
Comprehensive multi-disciplinary care standards/guidelines for DMD have been developed by the Centers for Disease Control and Prevention (CDC), and were published in two parts in The Lancet Neurology in 2010. To download the two articles in PDF format, go to the TREAT-NMD website: http://www.treat-nmd.eu/downloads/file/standardsofcare/dmd/lancet/the_diagnosis_and_management_of_dmd_lancet_complete_with_erratum.pdf
Read more about this topic: Duchenne Muscular Dystrophy
Famous quotes containing the word treatment:
“Judge Ginsburg’s selection should be a model—chosen on merit and not ideology, despite some naysaying, with little advance publicity. Her treatment could begin to overturn a terrible precedent: that is, that the most terrifying sentence among the accomplished in America has become, “Honey—the White House is on the phone.””
—Anna Quindlen (b. 1952)
“The motion picture made in Hollywood, if it is to create art at all, must do so within such strangling limitations of subject and treatment that it is a blind wonder it ever achieves any distinction beyond the purely mechanical slickness of a glass and chromium bathroom.”
—Raymond Chandler (1888–1959)
“I feel that any form of so called psychotherapy is strongly contraindicated for addicts.... The question “Why did you start using narcotics in the first place?” should never be asked. It is quite as irrelevant to treatment as it would be to ask a malarial patient why he went to a malarial area.”
—William Burroughs (b. 1914)