Sangamo Announces Treatment of First Patient in Phase 1/2 Clinical Trial Evaluating SB-318 Investigational in Genome Editing Therapy for MPS I

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Sangamo Announces Treatment of First Patient in Phase 1/2 Clinical Trial Evaluating SB-318 Investigational in Genome Editing Therapy for MPS I

The EMPOWERS Study is an open-label, ascending dose clinical trial designed to assess the safety, tolerability and preliminary efficacy of SB-318 in up to nine adult subjects with attenuated MPS I. The study is currently screening subjects at hospitals specializing

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10 Things to Know About Clinical Trials

Clinical trials have led to important medical discoveries like vaccines and treatments for cancer, heart disease, diabetes, and rare diseases. Without clinical trials, we wouldn’t have any of the medicines we take every single day. Knowing more about clinical trials

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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.