Update on the BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program

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Update on the BioMarin Sanfilippo Syndrome Type B (MPS IIIB) Clinical Development Program

An Update for Patient Associations and Families from BioMarin May 2018 We would like to update you about the status of our clinical development program in Sanfilippo B. The BioMarin program, which involves multiple centers around the world, consists of

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Stand on your own – Adult MPS patient meeting in Germany

This fellowship has now closed and is not accepting any more applications. Adult  MPS  patients  meeting STAND  ON  YOUR  OWN October  4  and  5,  2018 Maritim  Hotel  Gelsenkirchen,  Germany  For  the  first  time  MPS  Europe  is  organising  Adult  MPS  patients

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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