OTL-200 for Metachromatic Leukodystrophy Investigational New Drug Application Gets Approved
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OTL-200 for Metachromatic Leukodystrophy Investigational New Drug Application Gets Approved

  In a recent press release, gene therapy company Orchard Therapeutics ("Orchard") announced that the FDA approved Orchard's Investigational New Drug (IND) application for OTL-200. This gene therapy solution is…

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Natural History Study on Metachromatic Leukodystrophy To Use Innovative Methodology
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Natural History Study on Metachromatic Leukodystrophy To Use Innovative Methodology

As reported in Biospace, the National Organization for Rare Disorders (NORD) has just opened registration for patients to take part in a natural history study on metachromatic leukodystrophy (MLD), a…

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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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Defective Immune Cells Cause Rare Childhood Neurological Disorders
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Defective Immune Cells Cause Rare Childhood Neurological Disorders

A recent University of Sheffield news release explores findings which highlight a potential cause of leukodystrophies: immune cells. This group of rare childhood neurological disorders impacts the myelin sheath, 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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Rare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis

Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…

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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…

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