Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies 15
The report provides a snapshot of the global therapeutic landscape of
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )
Mucopolysaccharidosis III: molecular genetics and genotype-phenotype correlations.
Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with
mucopolysaccharidosis type I: results of a European consensus procedure.
Ethical and economic considerations of rare diseases in ethnic minorities: the case of
mucopolysaccharidosis VI in Colombia.
A snapshot of the global therapeutic scenario for
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ).
Rapid method for measuring arylsulfatase A and B in leucocytes as a diagnosis for sulfatidosis, mucosulfatidosis and
mucopolysaccharidosis VI.
Hunter syndrome is a genetic disorder classified as one of the lysosomal storage diseases, specifically
mucopolysaccharidosis II.
Approved products include Naglazyme (galsulfase) for
mucopolysaccharidosis VI, Kuvan (sapropterin dihydrochloride) for phenylketonuria, Aldurazyme (laronidase) for
mucopolysaccharidosis I and Firdapse (amifampridine phosphate) for the treatment of Lambert Eaton Myasthenic Syndrome.
The Morquio--Brailsford syndrome, known as
mucopolysaccharidosis type IV (MPS IV), is listed as a "rare disease" by the Office of Rare Diseases of National Institutes of Health, with a prevalence of 1/ 200 000-250 000 people.
Mucopolysaccharidosis (MPS) is a rare inherited biochemical disorder characterised by the accumulation of mucopolysaccharides (glycosaminoglycans) in various body tissues owing to insufficient amounts of the enzyme galactosamine sulphate (type A) and galactosidase (type B) to break them down.
Then, in November, the FDA warned of the potential for foreign particle contamination in all lots of the two aforementioned therapies plus Myozyme (alglucosidase alpha) for Pompe disease, Aldurazyme (laronidase) for
mucopolysaccharidosis type I and Thyrogen (thyrotropin alpha) for thyroid cancer.
