Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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Promising Interim Data in Phase 1/2 Hunter Syndrome Trial

Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…

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FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome
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FDA Grants Rare Pediatric Disease Designation to AVR-RD-05 for Hunter Syndrome

According to an article published in Yahoo, the FDA has recently granted the Rare Pediatric Disease designation to AVR-RD-05, a gene therapy for Hunter syndrome. This designation is reserved for…

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Natural Language Processing is Changing How We Research Rare Diseases
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Natural Language Processing is Changing How We Research Rare Diseases

Diagnosing Rare Diseases Diagnosing rare diseases is historically difficult. Physicians are taught to think about horses instead of zebras because horses are abundantly more common. For instance, if you're tired…

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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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New Reduced-Intensity Conditioning for HSCT Found Beneficial for 20 Rare Diseases

A recent study published in the journal Blood Advances has documented that hematopoietic stem cell transplantation (HSCT) is both safe and effective for children with many different kinds of inherited nonmalignant conditions…

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Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy
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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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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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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases
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India Turns to Enzyme Replacement Therapy as it Seeks to Address Rare Diseases

According to a story from health.economictimes.indiatimes.com, the country of India is currently beginning to take more steps in order to address the dire unmet medical need of its rare disease…

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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders
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Takeda Pharmaceutical Launches Trio of Enzyme Replacement Therapies in India to Treat Lysosomal Storage Disorders

According to a press release from Takeda Pharmaceutical Company published by For Press Release, the Company has launched a collection of enzyme replacement therapies for use in the treatment of…

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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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The Argument for Mandatory Newborn Screening and Government Subsidies for Rare Diseases in India

The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…

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