Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
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Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders

  According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…

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After Five Insurance Coverage Denials, the Next Appeal Brought a Young Patient Closer to Receiving Treatment for Metachromatic Leukodystrophy

  A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…

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4-Year-Old With Metachromatic Leukodystrophy Must Seek Out-of-State Treatment
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4-Year-Old With Metachromatic Leukodystrophy Must Seek Out-of-State Treatment

  Finding the correct doctor for treatment can be difficult, especially if one has a rare disease. Because few doctors specialize in rare diseases, they are often spread across the…

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OTL-200 Granted Accelerated Assessment for Treatment of Metachromatic Leukodystrophy (MLD)
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OTL-200 Granted Accelerated Assessment for Treatment of Metachromatic Leukodystrophy (MLD)

  The development of treatments and medicines for diseases can take a long time. These treatments come from many hours of research, studies, trials, and waiting for approval from the…

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Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising

According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…

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Proof of Concept Data Highlights Viability of Gene Therapy for Metachromatic Leukodystrophy
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Proof of Concept Data Highlights Viability of Gene Therapy for Metachromatic Leukodystrophy

According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc., recently announced the presentation of data which revealed that the company's proprietary virus vectors, which are…

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Experimental Drug for Metachromatic Leukodystrophy Gets Rare Pediatric Disease Designation
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Experimental Drug for Metachromatic Leukodystrophy Gets Rare Pediatric Disease Designation

According to a story from EPM Magazine, the pharmaceutical company Orchard Therapeutics received Rare Pediatric Disease Designation for its gene therapy drug candidate OTL-200. The therapy is in development for…

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