Article
Elosulfase alfa.
Drugs of today (Barcelona, Spain : 1998) - 1 Jul 2014
Haddley K
Abstract excerpt
Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome, is an inherited, lysosomal storage disorder caused by genetic mutations in N-acetylgalactosamine-6-sulfatase (GALNS) enzyme gene. GALNS is essential for breakdown of glycosaminoglycans. The disease is characterized by the early onset of severe skeletal dysplasia resulting in significant disability by the second decade of life. Until...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
