At present, there is no cure for the condition, and to date management of alpha mannosidosis has largely focused on treatment of the various symptoms that can be present1B.
Some of the main complications include
Each of these problems will have to be cared for by a specialist, in addition to an expert in rare metabolic diseases, and patients are typically seen by an ophthalmologist (eye doctor), an otolaryngologist (ear, nose and throat), hearing specialist and psychiatrist or psychologist, in addition to an orthopaedic specialist3B.
Ideally, the individual with alpha-mannosidosis will be followed by a team of medical specialists who can help determine the specific needs of each person and help suggest the most appropriate solutions. This may include, for example, the use of a hearing aid, educational interventions to improve social skills and even orthopaedic surgery to correct some skeletal abnormalities4B. Whatever interventions are needed, consider that treatment is always proactive, and aimed on preventing complications and treating symptoms of the condition, such as infections.
Today, newer treatments that address the cause of the disease are now being studied.
Most recently, in Europe, enzyme replacement therapy has been approved for use in adults, adolescents and children with mild–moderate forms of alpha-mannosidosis5B. In enzyme replacement therapy, the defective enzyme is replaced (in this case alpha-mannosidase) with a normal or more active form of the enzyme that is lacking.
Another type of therapy is called bone marrow transplantation (BMT) which may help donor cells repopulate the patient’s tissues and transfer normal enzyme to cells that make the incorrect form6B. However, while considered promising, the procedure may be associated with some risks7B.
Thus, while some therapies that have shown encouraging results, your doctor and team of specialists can help determine which treatment is most appropriate after careful assessment and evaluation of the individual with the disease.
The information on this website is intended only to provide knowledge of Alpha Mannosidosis disease health topics. This information should not be used in place of advice from your GP or other healthcare professional. If in doubt please contact your doctor for advice. This website has been produced by Chiesi Pharmaceuticals. The website has been developed in accordance with industry and legal standards to provide information for healthcare professionals and the general public about Alpha Mannosidosis disease health topics. Chiesi Pharmaceuticals makes every reasonable effort to include accurate and current information. However, the information provided in this website is not exhaustive.