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National MPS Society Annual Report 2015

Research Now: Gene Therapy for Sanfilippo Syndrome Moves Toward Clinical Trials


Study verifies human gene therapy in MPS I animal model


CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)


Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)


A Phase I/II, Randomized, Saftey and Ascending Dose Ranging Study of Intrathecal Idursulfase-IT Administered in Conjuction with Intravenous Elaprase in Pediatric Patients with Hunter Syndrome and Cognitive Impairment


Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

News Articles & Updates

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