Is there a treatment for alpha-mannosidosis?
Treatment for alpha-mannosidosis is mostly based on treating the different symptoms that people with the condition have. 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.
As of 2018, 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.
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).
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.