Beata Welk, Mirosław Szeligowski, Andrzej Stańczak

Choroby sieroce. Część I. Mukopolisacharydozy i preparaty stosowane w ich leczeniu

Rare diseases. Part 1. Mucopolysaccharidoses and preparations used in therapy. Rare diseases are serious chronic diseases that affect a small number of people compared to the general population. Mucopolysaccharidoses are both rare and genetic lysosomal storage diseases caused by the bodyís inability to produce specific enzymes. This paper provides information about MPS and drugs - laronidase and galsulfase, enzymes replacement therapies for MPS I and MPS VI.