Treatment for this disease is pretty limited, but there's some experimental treatment available. Laronidase is made possible by recombinant DNA technology. It has been indicated to treat MPS type 1. It has shown improvement in the patient's walking capacity and function. Exercising and range of motion (ROM) exercising have been shown to dramatically improve the patient's ability to maneuver limbs. Bone marrow transplantation, (BMT) can treat MPS conditions and Hurler syndrome. Those treated with BMT have been seen generally to have a longer lasting lifespan compared to those who have not been treated with BMT. There is also a very noticeable difference in the skeletal structure after this kind of treatment.
Genetic testing is available. |
Surgical treatment options.
Patients with this disease generally take on very uncommon, rarely used, and extremely dangerous surgeries. It's extremely dangerous, because there's a high number of patients who have died post-surgery. This is due to the heart and lung problems and diseases already present in the patient. Here are a list of all the treatment options available.
Corneal Clouding - This is only performed for severe cases, but there is not enough evidence to show if it is reliable long-term or not.
Valve Replacement - Mitral and aortic valves are replaced and adjusted into the body.
Tracheostomy - Surgery in the throat to manage patients with severe apnea (temporary cessation of breathing, especially during sleep) with good success.
Orthopedic Surgeries - These are surgeries pertaining to soft tissue and the bones. The procedures are generally performed on the hip, knee, and ankle. They make the bones progressively grow or are removed partly carefully.
Corneal Clouding - This is only performed for severe cases, but there is not enough evidence to show if it is reliable long-term or not.
Valve Replacement - Mitral and aortic valves are replaced and adjusted into the body.
Tracheostomy - Surgery in the throat to manage patients with severe apnea (temporary cessation of breathing, especially during sleep) with good success.
Orthopedic Surgeries - These are surgeries pertaining to soft tissue and the bones. The procedures are generally performed on the hip, knee, and ankle. They make the bones progressively grow or are removed partly carefully.
Recent Developments:
There are many treatements currently being tested right now to help patients with mucopolysaccharidosis. One of these are called Enzyme replacement therapy (ERT). This treatment replaces an enzyme in patients in whom that particular enzyme is deficient or absent. In this case, it will be one of the enzymes that the patient is missing which disables them to break down mucopolysaccharides. Some more current treatment options being tested are bone marrow transplantation (BMT) and umbilical cord blood transplantation (UCBT), which both have had limited success in treating the mucopolysaccharidoses. Both of theses treatments are used to get stem-cells, which scientists hope can help patients. But right now, BMT and UCBT are high-risk procedures and are usually performed only after family members receive extensive evaluation and counseling.