Researchers Fail to Find a Cure for MPS Through Genome Editing

The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…

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This Dad Climbed a Literal Mountain for His Daughter with Pompe Disease

The Malaysia Lysosomal Diseases Association (MLDA) was formed in 2011 by the hands of eight families. Their goal was to raise awareness and funds for those diagnosed with lysosomal storage…

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Cognitive Impairment, Testing, and Disease Burden in MPS II Discussed at Satellite Symposium

Today, Professor Elsa Shapiro and Dr. Christina Lampe presented "Cognitive Impairment in Patients with MPS II: From Disease Burden to Cognitive Testing" at Satellite Symposium, an event sponsored by Shire…

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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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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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Teen with Hunter Syndrome in Jeopardy Due to Clerical Error
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Teen with Hunter Syndrome in Jeopardy Due to Clerical Error

Some lucky people live their whole lives only taking medication intermittently, like pain killers when they have a headache or acute pain. Maybe they take an anti-inflammatory when they have…

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