Are there other organ or symptom specific treatments for alpha-mannosidosis?
Treatment for alpha-mannosidosis is mostly based on treating the different symptoms that people with the condition have. As of 2019, there is no FDA-approved systemic treatment for alpha-mannosidosis in the U.S. There are clinical trials in the United States that have studied or are studying the use of enzyme replacement therapy for alpha-mannosidosis.
A hematopoietic stem cell transplant (HSCT) can also be used to treat some of the symptoms of alpha-mannosidosis. These transplants can improve the levels of alpha-mannosidase in the body to treat some of the symptoms of the condition. A HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems that people with alpha-mannosidosis can have with their mental development and the brain. HCST has significant risks to the person undergoing the procedure and is not done on a routine basis. The risks and benefits of HSCT should be discussed with your doctor.
There are orthopedic surgeries that individuals with alpha-mannosidosis can have to relieve some of the pain and other complications from their bone problems. There are antibiotics and vaccinations available for people with alpha-mannosidosis that can prevent some of the infections that may be common for people with this condition. Hearing aids can be used to improve nerve related hearing loss, and glasses can be used to help with vision problems.
Depending on a person's specific symptoms of Alpha-mannosidosis, there may be other treatment methods that they might find helpful.
In January 2018, the European Medicines Agency (EMA) granted a marketing authorization under exceptional circumstances in the European Union for Lamzede (velmanase alfa, Chiesi Farmaceutici S.p.A.) to treat alpha-mannosidosis. Lamzede is a long-term intravenous enzyme replacement therapy for adults, adolescents, and children with mild to moderate forms of alpha-mannosidosis. The applicant still needs to complete an open label study to further study Lamzede's effectiveness in patients under six years of age, and to evaluate long-term effectiveness and safety of the treatment (details available from http://www.ema.europa.eu/docs/en_GB/document_library/Press_release/2018/01/WC500242401.pdf).
SOURCE: Emory University - Department of Human Genetics in collaboration with ThinkGenetic • https://www.thinkgenetic.com/diseases/mannosidosis-alpha-b-lysosomal/treatment/9932 • DATE UPDATED: 2019-05-30
European Medicines Agency. Press release: New enzyme replacement therapy to treat genetic disorder alpha-mannosidosis in children and adults. Retrieved February 14, 2018 from http://www.ema.europa.eu/docs/en_GB/document_library/Press_release/2018/01/WC500242401.pdf.