Abstract | INTRODUCTION: Despite being reported for the first time almost one century ago, only in the last few decades effective have treatments become available for the mucopolysaccharidoses (MPSs), a group of 11 inherited metabolic diseases that affect lysosomal function. These diseases are progressive, usually severe, and, in a significant number of cases, involve cognitive impairment. AREAS COVERED: EXPERT OPINION: The available treatments have resulted in improvements for several disease manifestations, but they still do not represent a cure for these diseases; thus, it is important to develop alternative methods to approach the unmet needs (i.e. bone disease, heart valve disease, corneal opacity, and central nervous system (CNS) involvement). The work in progress with novel approaches makes us confident that in 2017, when MPS will commemorate 100 years of its first report, we will be much closer to an effective cure for these challenging conditions.
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Authors | Roberto Giugliani, Andressa Federhen, Filippo Vairo, Cláudia Vanzella, Gabriela Pasqualim, Letícia Machado Rosa da Silva, Luciana Giugliani, Ana Paula Kurz de Boer, Carolina Fishinger Moura de Souza, Ursula Matte, Guilherme Baldo |
Journal | Expert opinion on emerging drugs
(Expert Opin Emerg Drugs)
Vol. 21
Issue 1
Pg. 9-26
( 2016)
ISSN: 1744-7623 [Electronic] England |
PMID | 26751109
(Publication Type: Journal Article, Review)
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Topics |
- Animals
- Cognition Disorders
(etiology, therapy)
- Disease Progression
- Drug Design
- Enzyme Replacement Therapy
(methods)
- Genetic Therapy
(methods)
- Humans
- Mucopolysaccharidoses
(complications, physiopathology, therapy)
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