Mucopolysaccharidosis - Treatment

Treatment

Currently there is no cure for these disorders. Medical care is directed at treating systemic conditions and improving the person's quality of life. Physical therapy and daily exercise may delay joint problems and improve the ability to move.

Changes to the diet will not prevent disease progression, but limiting milk, sugar, and dairy products has helped some individuals experiencing excessive mucus.

Surgery to remove tonsils and adenoids may improve breathing among affected individuals with obstructive airway disorders and sleep apnea. Sleep studies can assess airway status and the possible need for nighttime oxygen. Some patients may require surgical insertion of an endotrachial tube to aid breathing. Surgery can also correct hernias, help drain excessive cerebrospinal fluid from the brain, and free nerves and nerve roots compressed by skeletal and other abnormalities. Corneal transplants may improve vision among patients with significant corneal clouding.

Enzyme replacement therapy (ERT) are currently in use or are being tested. Enzyme replacement therapy has proven useful in reducing non-neurological symptoms and pain. Currently BioMarin Pharmaceutical produces enzyme replacement therapies for MPS type I and VI. In July 2006, the United States Food and Drug Administration approved a synthetic version of I2S produced by Shire Pharmaceuticals Group, called Elaprase, as a treatment for MPS type II (Hunter syndrome).

Bone marrow transplantation (BMT) and umbilical cord blood transplantation (UCBT) have had limited success in treating the mucopolysaccharidoses. Abnormal physical characteristics, except for those affecting the skeleton and eyes, may be improved, but neurologic outcomes have varied. BMT and UCBT are high-risk procedures and are usually performed only after family members receive extensive evaluation and counseling.

For information on clinical trials visit Clinical Trials Search

Read more about this topic:  Mucopolysaccharidosis

Famous quotes containing the word treatment:

    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)

    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)

    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)