Study: Idursulfase Therapy is a Safe Treatment for Hunter Syndrome (MPS II)
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Study: Idursulfase Therapy is a Safe Treatment for Hunter Syndrome (MPS II)

  A recent meta-analyses of clinical studies and case reports that examined the safety of idursulfase therapy, which is an enzyme therapy, has shown that this form of treatment is…

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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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Treatment for Children with Hunter Syndrome Set for Clinical Trial
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Treatment for Children with Hunter Syndrome Set for Clinical Trial

REGENXBIO announced last month that the Investigational New Drug application (IND) is active for a Phase I/II clinical trial of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II)…

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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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First Patient with MPS II Receives Brand New Gene Therapy
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First Patient with MPS II Receives Brand New Gene Therapy

Sangamo Therapeutics shared some big news about the search for Mucopolysaccharidosis II (MPS II) treatment. They reported that they have, for the very first time, used experimental in vivo gene therapy to treat…

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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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It’s a Hunter Syndrome Pediatric Indication Swap-Meet!
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It’s a Hunter Syndrome Pediatric Indication Swap-Meet!

Okay, I’m going to be perfectly honest: I don’t quite know what to make of this. On the one hand, you have some excellent news for people living with mucopolysaccharidosis…

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Sharing Jonathan and Bryce’s Hunter Syndrome Stories Helps Us All
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Sharing Jonathan and Bryce’s Hunter Syndrome Stories Helps Us All

Everyone has a story. Every person has something that they can share. Reading about another person’s story can help you and your family cope with Hunter syndrome. Also, learning about…

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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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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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