Dec 18, 2019
REGENXBIO Announces Interim Data from Phase I/II Trial of RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
Dec 04, 2019
Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 - 2026
Oct 23, 2019
Allievex Completes Series A Financing Led by Pappas Capital and Novo Holdings
Jul 03, 2019
Mucopolysaccharidosis (MPS) Treatment Market: Global Industry Analysis 2014-2018 and Opportunity Assessment 2019-2029
Apr 03, 2019
Clinigen K.K. and GC Pharma Announce Exclusive Licensing Agreement in Japan for Hunterase (Idursulfase-beta) ICV, Hunter Syndrome Drug
Feb 07, 2019
Sangamo Announces Interim Results Of Phase 1/2 CHAMPIONS Study Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II Treated With SB-913
Sep 05, 2018
Sangamo Announces 16 Week Clinical Results Including Reductions In Glycosaminoglycans In Phase 1/2 Trial Evaluating SB-913, A Zinc Finger Nuclease Genome Editing Treatment For MPS II (Hunter Syndrome)
Aug 17, 2018
2018 Mucopolysaccharidosis Disorders Drug Development Pipeline Review
Aug 10, 2018
First Patient Dosed in Phase 1/2 Study Evaluating SOBI003 for Treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA)
Aug 02, 2018
ArmaGen Receives U.S. Orphan Drug Designation for AGT-184
Jul 27, 2018
Sangamo To Host Educational Webcast With Hunter Syndrome (MPS II) Expert, Joseph Muenzer, M.D., Ph.D.
Jul 10, 2018
ArmaGen's AGT-181 52-Week Phase 1/2 Proof-of-Concept Study Results Published in Orphanet Journal of Rare Diseases
May 15, 2018
REGENXBIO Joins the Mucopolysaccharidosis Community to Advance Research and Innovation
May 02, 2018
REGENXBIO Receives FDA Fast Track Designation for RGX-121 Gene Therapy for the Treatment of Mucopolysaccharidosis Type II
Apr 04, 2018
Sangamo Announces Publication In Molecular Therapy Of Preclinical Study Data From MPS II In Vivo Genome Editing Program
Feb 08, 2018
ArmaGen's AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe MPS I
Feb 06, 2018
Sangamo Therapeutics Presents Initial Safety Data from CHAMPIONS Genome Editing Study for MPS II at WORLDSymposium
Nov 30, 2017
ArmaGen's AGT-181 Granted Fast Track Designation for the Treatment of Hurler Syndrome (MPS I)
Sep 06, 2017
BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at 13th International Congress of Inborn Errors of Metabolism (ICIEM) 2017
Jun 15, 2017
Mucopolysaccharidosis III - Pipeline Review, H1 2017