Oct 23, 2019
Allievex Completes Series A Financing Led by Pappas Capital and Novo Holdings
Feb 07, 2019
Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I
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
Apr 04, 2018
Sangamo Announces Publication In Molecular Therapy Of Preclinical Study Data From MPS II In Vivo Genome Editing Program
Mar 14, 2018
Jupiter Orphan Therapeutics Announces Submission of Investigational New Drug Application (IND) For Treatment Of Mucopolysaccharidosis Type-I
Feb 08, 2018
ArmaGen's AGT-181 Demonstrates Neurocognitive Benefit in Children with Severe MPS I
Feb 07, 2018
BioMarin Presents Interim Data of Phase 1/2 Study of BMN 250 for Treatment of Sanfilippo B Syndrome (MPS IIIB) at WORLDSymposium(TM) 2018
Feb 05, 2018
Jupiter Orphan Therapeutics in Process to Submit IND for MPSI and Other Indications
Dec 07, 2017
Sangamo Announces European Medicines Agency Recommendation Of Orphan Medicinal Product Designation For Investigational Genome Editing Treatments For MPS I And MPS II
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