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:
“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)
“[17th-century] Puritans were the first modern parents. Like many of us, they looked on their treatment of children as a test of their own self-control. Their goal was not to simply to ensure the child’s duty to the family, but to help him or her make personal, individual commitments. They were the first authors to state that children must obey God rather than parents, in case of a clear conflict.”
—C. John Sommerville (20th century)
“James’s great gift, of course, was his ability to tell a plot in shimmering detail with such delicacy of treatment and such fine aloofness—that is, reluctance to engage in any direct grappling with what, in the play or story, had actually “taken place”Mthat his listeners often did not, in the end, know what had, to put it in another way, “gone on.””
—James Thurber (1894–1961)