Mepsevii for MPS VII Reimbursement Approval Available in Spain
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Mepsevii for MPS VII Reimbursement Approval Available in Spain

According to a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…

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Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding
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Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding

According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…

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Sanofi is Making Strides in Lysosomal Storage Disease Research

Sanofi There are an estimated 350 million rare disease patients across the world. Many conditions still don't have treatments. However, there are scientists dedicating their lives solely to this endeavor.…

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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit

  On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…

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Inventiva Will Present Findings from Maroteaux-Lamy Syndrome Trial at Upcoming Conference
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Inventiva Will Present Findings from Maroteaux-Lamy Syndrome Trial at Upcoming Conference

According to a story from globenewswire.com, the biopharmaceutical company Inventiva is scheduled to present the findings from a recent phase IIa clinical trial at the upcoming 16th Annual WorldSymposium™ which is…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?
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Will Therapies for Neurological Disorders Bring Forth a New Era for Biotech?

According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…

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Study: Odiparcil Shows Positive Results in Treatment of Mucopolysaccharidosis Type VI (MPS VI)
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Study: Odiparcil Shows Positive Results in Treatment of Mucopolysaccharidosis Type VI (MPS VI)

Inventiva has recently completed Phase IIa of their clinical trial of odiparcil, a drug made for the treatment of mucopolysaccharidosis type VI (MPS VI). Iventiva is very excited about the…

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Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy

  East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…

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A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far
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A Proof-of-Concept Study for an Experimental Hurler Syndrome Treatment is Going Well so Far

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…

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The Isaac Foundation has Turned to Television to Get Their Mucopolysaccharidosis Message Heard

The Isaac Foundation The Isaac Foundation was established by a man named Andrew McFadyen. He created the organization in 2007 in honor of  his son who is diagnosed with the rare…

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