Tracheostomy in
mucopolysaccharidosis type {II} (Hunter's syndrome) Int.
The impact of Hunter syndrome (
mucopolysaccharidosis type II) on health-related quality of life.
Mucopolysaccharidosis IV A: assignment of the human N-acetylgalactosamine-6-sulfate sulfatase (GALNS) gene to chromosome 16q24.
Van Den Biggelaar et al., "Severe tracheal and bronchial collapse in adults with type II
mucopolysaccharidosis," Orphanet Journal of Rare Diseases, vol.
Malm, and HOS Investigators, "Hearing loss in patients with
mucopolysaccharidosis II: data from HOS-the Hunter Outcome Survey," Journal of Inherited Metabolic Disease, vol.
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies 11
Mucopolysaccharidosis type IV (MPS type IV) is a rare disorder which presents with a number of musculoskeletal defects.
Abbreviations CMV: Cytomegalovirus CNS: Central nervous system CP: Cortical plate, cerebral Hb: Hemoglobin HE: Hematoxylin-Eosin (staining) HF: Hydrops fetalis LSD: Lysosomal storage diseases MPS:
Mucopolysaccharidosis NIHF: Nonimmune hydrops fetalis PAS: Periodic Acid-Schiff (staining) PAS-D: Periodic Acid-Schiff-diastase/amylase (staining) PCR: Polymerase chain reaction PG: Polyglucosans PGB: Polyglucosan bodies PGBD: Polyglucosan Body Disease(s)/Polyglucosan Body Disorder(s) APGBD: Adult Polyglucosan Body Disease WM: White matter, cerebral.
Enzymereplacement therapy in
mucopolysaccharidosis I.
The report provides comprehensive information on the therapeutics under development for
Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.
Molecular genetics of
mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications.
Dr Saqib Ansari said, The median survival for
mucopolysaccharidosis I (MPS I) patients is less than 11.6 years if not treated in an early age.