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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May 15th is Mucopolysaccharidosis (MPS) Awareness Day: Spreading Rare Disease Awareness
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May 15th is Mucopolysaccharidosis (MPS) Awareness Day: Spreading Rare Disease Awareness

May 15th is recognized each year as Mucopolysaccharidosis (MPS) Awareness Day. The goal of this day is to help spread awareness about this group of diseases among the general public…

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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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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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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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First Patients Recruited in Maroteaux-Lamy Syndrome Biomarker Study
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First Patients Recruited in Maroteaux-Lamy Syndrome Biomarker Study

According to a story from Benzinga, the biopharmaceutical company Inventiva has recently announced that it has recruited the first patients in a study of biomarkers in Maroteaux-Lamy syndrome. The study…

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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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IND Accepted for an Experimental Sanfilippo Syndrome Treatment
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IND Accepted for an Experimental Sanfilippo Syndrome Treatment

According to a story from forextv.com, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND)…

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Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application
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Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application

According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…

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Rare Disease Patients in Uttar Pradesh Forced to Abandon Treatment Amid Funding Crisis
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Rare Disease Patients in Uttar Pradesh Forced to Abandon Treatment Amid Funding Crisis

According to a publication from ET Healthworld, some 21 rare disease patients in India's northern state of Uttar Pradesh are still awaiting governmental financial assistance to which they are legally…

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Patient Recruitment Completed for Phase 2 Study of Mucopolysaccharidosis VI Drug Candidate

According to a press release from the French biotechnology company Inventiva, the company has completed recruitment for a phase 2 clinical trial evaluating its experimental mucopolysaccharidosis VI drug, odiparcil. About…

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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation
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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…

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China Approves Mucopolysaccharidosis Type IVA Drug As Part of Accelerated Review Program
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China Approves Mucopolysaccharidosis Type IVA Drug As Part of Accelerated Review Program

According to a publication from BioPortfolio, China's National Medical Products Administration (NMPA — similar to the American FDA) recently approved BioMarin's mucopolysaccharidosis type IVA drug Vimizim for use in the…

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First Treatment in China for Morquio A Syndrome Earns Approval
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First Treatment in China for Morquio A Syndrome Earns Approval

According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…

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Orchard Therapeutics Acquires Global Licensing Agreement for Experimental Mucopolysaccharidosis Drug

According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…

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Experimental MPS VI Drug Granted Rare Pediatric Disease Designation

A recent press release from the French biopharmaceutical company Inventiva announced that the Food and Drug Administration (FDA) had granted the company's experimental mucopolysaccharidosis VI (MPS VI) drug odiparcil rare…

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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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