Mucopolysaccharidosis Type VII
Treatment
Is there a treatment(s) for Mucopolysaccharidosis Type VII?
Currently there is one Food and Drug Administration (FDA) approved treatment for Mucopolysaccharidosis Type VII (MPS VII): MEPSEVII™ (vestronidase alfa). MEPSEVII™ is an enzyme replacement therapy (ERT) made by Ultragenyx Pharmaceutical Inc. The goal of ERT is to replace the enzyme missing in individuals with MPSVII with an artificial or recombinant beta-glucuronidase. ERT helps to breakdown incorrectly stored materials in the cells (the glycosaminoglycans (GAGs) dermatan sulfate, chondroitin sulfate and heparan sulfate). Patients getting ERT usually visit an infusion center every 2 weeks and receive the enzyme by an intravenous (IV) infusion. When began early in the course of the disease, replacing the enzyme helps slow the progression of the disease, reduces complications, and may even prevent long-term complications.
In some patients another treatment option is a hematopoietic stem cell transplant (HSCT) or bone marrow transplant. HSCT can also be used to treat some of the symptoms of MPS VII, but HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems that people with MPS VII can have with their mental development and the brain. The bone marrow produces blood cells and the theory behind the transplant is to remove the affected patient's bone marrow and replace it with the bone marrow from someone who doesn't have MPS VII. The white blood cells produced from this new bone marrow will be able to produce some β-glucuronidase enzyme. HCST has significant risks to the person undergoing the procedure. The risks and benefits of HSCT should be discussed with a doctor experienced in HSCT for MPS VII.
Beyond ERT, ongoing medical care is based on treating a person's different symptoms. MPS VII affects many systems in the body. Depending on the symptom to be treated, many different specialists can be involved, such as cardiologists (heart doctors), pulmonologists (breathing/lung doctors), medical geneticists (genetics doctors), ophthalmologists (eye doctors), audiologists (hearing specialists), otolaryngologists (or ENTs; ear, nose and throat doctors), neurologists (neurologists), and surgeons for any other health problems that may require surgical treatment. For example, there are orthopedic surgeries that individuals with MPS VII can have to relieve some of the pain and other complications from their bone problems. There are also some surgeries to remove the tonsils and adenoids that can help with some of the breathing problems like sleep apnea (where breathing stops or is very shallow during sleep, often caused by a narrow airway). Certain diet changes can be used in an attempt to treat some of the diarrhea and constipation experienced by some people with MPS VII.
References
- http://mpssociety.org/wp-content/uploads/2011/07/MPS_VII_2008.pdf
- https://rarediseases.org/rare-diseases/sly-syndrome/
- Ultragenyx Pharmaceutical, "How Do I Get Started/" accessed 4/19/2018 http://www.mepsevii.com/getting-started-on-mepsevii/getting-started/
More Treatment Content
What other things can be done to help the symptoms of Mucopolysaccharidosis Type VII?
Are there other organ or symptom specific treatments for Mucopolysaccharidosis Type VII?
What other things can be done to help the symptoms of Mucopolysaccharidosis Type VII?
Beyond enzyme replacement therapy with MEPSEVII™ (vestronidase alfa), medical care is mostly based on treating a person's different symptoms. For some individuals, a hematopoietic stem cell transplant (HSCT) or bone marrow transplant may be used to treat some of the symptoms of MPS VII; however, HSCT does not usually improve the problems with the bones, problems with the eyes, or the problems that people with MPS VII can have with their mental development and the brain. The bone marrow produces blood cells and the theory behind the transplant is to remove the affected patient's bone marrow and replace it with the bone marrow from someone who doesn't have MPS VII. The white blood cells produced from this new bone marrow will be able to produce some β-glucuronidase enzyme. HCST has significant risks to the person undergoing the procedure. The risks and benefits of HSCT should be discussed with a doctor experienced with HSCT in patients with MPS VII.
MPS VII affects many systems in the body. Depending on the symptom to be treated, many different specialists can be involved, such as cardiologists (heart doctors), pulmonologists (breathing/lung doctors), medical geneticists (genetics doctors), ophthalmologists (eye doctors), audiologists (hearing specialists), otolaryngologists (or ENTs; ear, nose and throat doctors), neurologists (neurologists), and surgeons for any other health problems that may require surgical treatment. For example, there are orthopedic surgeries that individuals with MPS VII can have to relieve some of the pain and other complications from their bone problems. There are also some surgeries to remove the tonsils and adenoids that can help with some of the breathing problems like sleep apnea (where breathing stops or is very shallow during sleep, often caused by a narrow airway). Certain diet changes can be used in an attempt to treat some of the diarrhea and constipation that can affected people with MPS VII.
References
- http://mpssociety.org/wp-content/uploads/2011/07/MPS_VII_2008.pdf
Are there other organ or symptom specific treatments for Mucopolysaccharidosis Type VII?
Mucopolysaccharidosis type VII (MPS VII) affects many systems in the body. Depending on the symptom to be treated, many different specialists can be involved, such as cardiologists (heart doctors), pulmonologists (breathing/lung doctors), medical geneticists (genetics doctors), ophthalmologists (eye doctors), audiologists (hearing specialists), otolaryngologists (or ENTs; ear, nose and throat doctors), neurologists (neurologists), and surgeons for any other health problems that may require surgical treatment. For example, there are orthopedic surgeries that individuals with MPS VII can have to relieve some of the pain and other complications from their bone problems. There are also some surgeries to remove the tonsils and adenoids that can help with some of the breathing problems like sleep apnea (where breathing stops or is very shallow during sleep, often caused by a narrow airway). Certain diet changes can be used in an attempt to treat some of the diarrhea and constipation that people with MPS VII can have.
References
- http://mpssociety.org/wp-content/uploads/2011/07/MPS_VII_2008.pdf
- https://rarediseases.org/rare-diseases/sly-syndrome/
