Press release on stem cell gene therapy approach for MPSII Hunter disease

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Press release on stem cell gene therapy approach for MPSII Hunter disease

A team at The University of Manchester have developed a novel stem cell gene therapy approach to treat children with a devastating genetic disease. The approach is currently being developed for clinical trial in patients with the disease. Although there

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Latest results on MPS I Clinical trial ArmaGen AGT-181: Investigational Therapy for the Treatment of Patients with Hurler Syndrome

ArmaGen’s AGT-181 52-Week Phase 1/2 Proof-of-Concept Study Results Published in Orphanet Journal of Rare Diseases Novel Fusion Protein Demonstrates Neurocognitive Benefit in Children with Severe Mucopolysaccharidosis Type I (MPS I, or Hurler Syndrome), with Favorable Safety Profile Data Validate ArmaGen’s Platform

Next stage for new Sanfilippo type C gene therapy

At the University of Manchester scientists have developed a new gene therapy they hope will treat children with Sanfilippo disease type C , and plan to take it to clinical trial in the near future. US-based biotech Phoenix Nest Inc.

Bob Stevens and family on BBC Three Counties Radio

Listen to Bob and his family telling their story on BBC Three Counties Radio on Sunday 24 June.

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Regenxbio receives FDA fast track designation for RGX-111 gene therapy for the treatment of MPS I

Regenxbio have announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RGX-111. RGX-111 is a novel, one-time investigational treatment for Mucopolysaccharidosis Type I (MPS I), that is designed to deliver the human iduronidase (IDUA) gene directly