Article
Hematopoietic Disorders, Renal Impairment and Growth in Mucopolysaccharidosis-Plus Syndrome.
International journal of molecular sciences - 23 May 2022
Sofronova Viktoriia, Iwata Rina, Moriya Takuya, Loskutova Kiunniai, Gurinova Elizaveta, Chernova Mairanush, Timofeeva Anastasia, Shvedova Anna, Vasilev Filipp, Novgorodova Saina, Terawaki Seigo, Moriwaki Takahito, Sukhomyasova Aitalina, Maksimova Nadezhda, Otomo Takanobu
Abstract excerpt
Mucopolysaccharidoses (MPS) are rare lysosomal storage disorders (LSD) characterized by the excessive accumulation of glycosaminoglycans (GAG). Conventional MPS, caused by inborn deficiencies of lysosomal enzymes involved in GAG degradation, display various multisystemic symptoms-including progressive neurological complications, ophthalmological disorders, hearing loss, gastrointestinal and hepatobiliary issues,...
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