|Report Code: PP10145||Report Type: Indication Pipeline Reports||Available format:|
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a condition that affects various parts of the body and occurs almost exclusively in males. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals. At birth, individuals with MPS II do not display any symptoms of the condition. Later, between the age of 2 to 4 years, features like full lips, large rounded cheeks, broad nose, and an enlarged tongue (macroglossia) are seen in them. The vocal cords also enlarge, which result in deep and hoarse voice. Narrowing of the airway causes frequent upper respiratory infections and short pauses in breathing during sleep (sleep apnea). As this disorder progresses, individuals need medical assistance to keep their airway open. Most people with this disorder develop hearing loss and have recurrent ear infections.
The drug candidates of MPS II pipeline include, but are not limited to, AGT-182, JR-141 and SB-913. Further companies like ArmaGen Inc., JCR Pharmaceuticals Co. Ltd., and Sangamo Therapeutics Inc. are also involved in the pipeline for MPS II therapeutics.