General: Currently, there is no known cure for any form of mucopolysaccharidosis (MPS), but there are treatments available to manage symptoms, prevent complications, and improve quality of life. Physical therapy may be helpful in the prevention and treatment of joint pain and stiffness that is common in all types of MPS.
Bone marrow transplant: Some types of MPS have had limited success with bone marrow transplantation. The use of bone marrow transplantation may be beneficial when performed at an early age in MPS I. A bone marrow transplant may provide cells that produce normal, functioning enzymes that may be otherwise deficient in MPS. The use of early bone marrow transplants may help to delay intellectual disability (formerly mental retardation) and improve other symptoms, but does not reverse damage that has already happened. A proper donor must be found to perform a bone marrow transplant, and this procedure is considered high-risk due to possible complications.
Enzyme replacement: Recombinant enzyme products are available for the treatment of specific types of MPS. Depending on the enzyme that is deficient or missing, the appropriate enzyme replacement may help in reducing some of the symptoms associated with whatever disease is being treated. Research has shown that neurological symptoms and pain may improve with enzyme replacement therapy.
Physical therapy: Patients with MPS may benefit from seeing a physical therapist. A physical therapist can help patients maintain muscle tone and keep muscles strong and flexible through performing different exercises. A speech therapist may help a patient retrain the tongue and facial muscles to improve speech.
Surgery: Various types of surgery have been performed depending on the symptom being treated. For patients with sleep apnea and obstructive airway disease, the removal of tonsils and adenoids may be beneficial in improving breathing. Corneal transplants may be useful for treating patients with corneal clouding. Surgery has also been used to relieve compression on nerves and the spinal cord, repair hernias, fix heart abnormalities, and to place shunts to allow for drainage of cerebrospinal fluid.
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