MPS VII(mucopolysaccharide diseases type VII) is a rare genetic disease, there are less than 150 patients in the world.The clinical manifestation of MPS VII is sexual disease, disease development can affect the most tissues and organs.The characteristics of MPS VII is widely different between patients, but most of the patients have various skeletal abnormalities, these anomalies as the growth of the age and become more evident, such as short stature.The affected person may also have abnormal heart valves, liver and spleen enlargement, airway stenosis, which can lead to lung infection and difficulty breathing.
In Nov 15,2017,the FDA approved Mepsevii (vestronidase alfa - VJBK) inherited metabolic disease - glycosaminoglycan type VII (MPSVII) treatment in children and adults, the enzyme replacement therapy is complete developed by biopharmaceutical company Ultragenyx Pharmaceutical which focused on rare diseases treatment, this is the first treatment approved by the FDA approved for Ultragenyx.
MPS VII is composed of beta glucuronic acid caused by glycosidase lack of lysosomal storage disorder, lead to abnormal accumulation of toxic substances in the body cells. Mepsevii therapy achieved therapeutic effect by replace the missing beta glucuronic acid glycosidase.The effectiveness and safety of the enzyme replacement therapy has been through clinical trials and involved in 23 patients with five months to 25 years into the set of solutions.
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