National MPS Society Annual Report 2015

https://indd.adobe.com/view/df1f388c-7542-490e-9ef1-43f2233a7225

​

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

http://www.nationwidechildrens.org/medical-professional-publications/research-now-gene-therapy-for-sanfilippo-syndrome-moves-toward-clinical-trials?contentid=106129

 

Study verifies human gene therapy in MPS I animal model

http://www.news-medical.net/news/20160729/Study-verifies-gene-therapy-in-animal-model-of-MPS-I.aspx

 

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

http://insight.jci.org/articles/view/86696

 

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

https://clinicaltrials.gov/ct2/show/NCT02055118?term=NCT02055118&rank=1

 

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

http://www.shiretrials.com/-/media/files/clinical%20trials/clinicaltrialsen/clinical%20study%20reports/shire-hgt-hit-045-clinical-study-report-redact.pdf

 

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

https://clinicaltrials.gov/ct2/show/results/NCT00607386?sect=X301256#evnt