This document summarizes an article about a recombinant form of human N- acetylgalactosamine 4-sulfatase used to treat mucopolysaccharidosis type V, a lysosomal storage disorder. The treatment called galsulfase has shown positive results in patients, improving mobility. The document also analyzes different drug therapies for lysosomal disorders and discusses gene therapy as an alternative treatment, noting disadvantages like not being able to treat all pathology sites. It concludes that to benefit from therapies, patients need to be identified as early as possible, preferably as newborns.

1. Third Seminar: Against all Odds: Protein Crystallography and Graduate School
By Yadilette Rivera
The article fresh from the pipelinegalsulfase talks about a recombinant form of human N-
acetylgalactosamine 4-sulfatase for the treatment of the mucopolysaccharidosis type V.
This is a lysosomal storage disorder due to a defect in the enzymes that degrade the
mucopolysaccharides or glycosaminoglycans. And one of the symptoms that this disease
presents is that the person tends to be dwarf because his bones cannot developed well.
The article explain a treatment with galsulfase that have been tested with patients having
this disease and they have shown positives results improving their mobility. This paper
presents an analysis of different drugs to treat the lysosomal disorder. This part exposes
the gene therapy that could be an alternative to treat this disorder. Also, presents the
disadvantages of these therapies such as "the ability to treat all sites of pathology", "the
ability to provide a single-step therapy to continuously provide enzyme to all sites of
pathology", and "the ability to predict the presence and nature of irreversible pathology".
It concludes saying that to obtain the therapy benefits patients have to be identified as
early as possible, preferable when are newborn.