MPS I TREATMENT OPTIONS

MPS I is caused by genetic changes in the IDUA (alpha-L-iduronidase) gene, which lead to a lack of the IDUA enzyme activity.

MPS I may require a number of different treatments, which may only address signs, symptoms, complications, and not the underlying cause. Aldurazyme (laronidase) is an enzyme replacement therapy that replaces the deficient enzyme to help break down accumulated GAGs, which are the cause of the signs and symptoms of MPS I.

CPAP = continuous positive airway pressure; FDA = food and drug administration; GAGs, glycosaminoglycans; IDUA = alpha-L-iduronidase; MPS I = mucopolysaccharidosis type I