MPS VI

MPS VI

Maroteaux-Lamy disease

An autosomal recessive condition (OMIM:253200) caused by arylsulfatase B deficiency.

Clinical findings
Coarse facies, corneal clouding, progressive dysostosis multiplex, hepatomegaly.
 
Management
Bone transplant; enzyme replacement therapy with galsulfase (Naglazyme) improves growth and joint movement, but is extremely expensive ($350K/year).
References in periodicals archive ?
MPS VI patients typically show dysostosis multiplex, a specific radiologic expression involving the skull, thorax, pelvis, hands and spine.
Therapies are available for only 6 LSDs - Gaucher, Pompe, Fabry, Mucopolysaccharidosis (MPS) I (Hurler syndrome), MPS II (Hunter Syndrome) and MPS VI (MaroteauxLamy)," said Prof.
Currently therapies are available for only 6 LSDs - Gaucher, Pompe, Fabry, Mucopolysaccharidosis (MPS) I (Hurler syndrome), MPS II (Hunter Syndrome) and MPS VI (MaroteauxLamy).
Ha onze tipos de MPS, mas medicamentos para apenas tres delas, a MPS I (laronidase), a MPS II (idursulfase) e a MPS VI (galsulfase).
Atlantoaxial instability with resultant myelopathy and spastic quadriparesis has been described in both, MPS IV and MPS VI.
This acquisition of the intellectual property includes patents related to the purified form of Naglazyme and the method of using the enzyme in the treatment of MPS VI, which expire between 2022 and 2023.
Naglazyme was developed to supplement the deficient enzyme in MPS VI by providing an exogenous enzyme in a therapeutic approach known as enzyme replacement therapy (ERT).
BPPS currently provides support for MPS VI patients treated with Naglazyme, and a similar support program will be available for PKU patients.
As the first drug approved for MPS VI, the FDA and EC have both designated Naglazyme as an orphan drug, conferring seven years of market exclusivity in the United States and 10 years of market exclusivity in the European Union.
Therapies, such as bone marrow or hematopoietic stem-cell transplantation and enzyme replacement therapy (ERT), are currently available for several LSDs, including MPS I (5, 6), MPS VI (7), Gaucher disease (8), and Fabry disease (9,10).
BioMarin will be positioned to commercialize pediatric products itself in the United States Having a commercial infrastructure enables BioMarin to launch approved, internally-developed pediatric products by itself in the United States Aryplase, in development for the treatment of MPS VI, completed Phase 3 testing recently and if the results, which are expected this quarter, are positive, a regulatory filing for approval in the United States and Europe will be submitted in the fourth quarter of 2004.
BioMarin is currently conducting a Phase 3 trial of Aryplase(TM) for MPS VI and, pending positive results, plans to file a biologics license application in the fourth quarter of 2004.