Mucopolysaccharidosis Type VII (Sly syndrome) is an inborn error of metabolism characterized by a deficiency of the enzyme b-glucuronidase, progressively affecting most tissues and organs. Some affected patients have do not survive infancy while others have developmental delay and progressive intellectual disability. Giving relief to such patients, FDA has recently approved Vestronidase alfa for the treatment of this rare disorder. Previously, there was no approved treatment option for Mucopolysaccharidosis Type VII (MPS VII).
Most patients have progressive skeletal abnormalities including short stature. Affected individuals can also develop heart valve abnormalities, enlarged liver and spleen, and narrowed airways leading to lung infections and troubled breathing. The life expectancy of individuals with MPS VII depends on the severity of symptoms. Vestronidase alfa is an enzyme…
