Satellite Symposium Discusses Long-Term Outcomes for MPS IVA and VI Patients
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Satellite Symposium Discusses Long-Term Outcomes for MPS IVA and VI Patients

Yesterday, a group of experts presented "Changing lives: Long-term outcomes of MPS IVA & VI patients" at the Satellite Symposium sponsored by BioMarin. The program began with insights on the…

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Project Alive Paves the Way for Gene Therapy Research That Could Save Children With Hunter Syndrome
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Project Alive Paves the Way for Gene Therapy Research That Could Save Children With Hunter Syndrome

Gene therapy is on the rise, and it just might be exactly what many children suffering from genetic disorders need to save their lives. Today reported that an amazing organization…

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New Drug For Mucopolysaccharidosis Gets FDA Approval For Study
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New Drug For Mucopolysaccharidosis Gets FDA Approval For Study

The FDA approved a human study to be conducted by Swedish Orphan Biovitrum AB to treating mucopolysaccharidosis type lllA patients with a new drug called SOB1003, reported European Pharmaceutical Review. Not only…

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The First Treatment in History for Mucopolysaccharidosis VII Was Just Approved by the FDA
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The First Treatment in History for Mucopolysaccharidosis VII Was Just Approved by the FDA

According to a story from EParent, the pharmaceutical company Ultragenyx released a statement confirming that the company's product MEPSEVII had received approval for public use by the Food and Drug…

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Investigational New Drug Application for MPS II Gene Therapy
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Investigational New Drug Application for MPS II Gene Therapy

 REGENXBIO Inc., a pharamaceutical company specializing in gene therapy, just shared that it launched a Investigational New Drug application to conduct Phase I/II trials on the new therapy they're developing,…

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Is This What the MPS Research Community Been Missing?
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Is This What the MPS Research Community Been Missing?

Recent findings from research done at the University of Pennslyvania indicate a new biomarker associated with a group of rare metabolic diseases called mucopolysaccharidoses (MPS). A biomarker is a measurable…

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Catch it Now! High Five for the Little Ones Supporting Hunter Syndrome
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Catch it Now! High Five for the Little Ones Supporting Hunter Syndrome

As the saying goes, “Many hands make light work.” Know what’s even sweeter? When those hands are little—little tiny hands that finger paint, swing on the playground, and play monster…

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