Mucopolysaccharidosis News and Research

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Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans - long chains of sugar carbohydrates in each of our cells that help build bone, cartilage, tendons, corneas, skin and connective tissue. Glycosaminoglycans (formerly called mucopolysaccharides) are also found in the fluid that lubricates our joints.
Novel drug may be the answer in battling resistance in aggressive breast cancer

Novel drug may be the answer in battling resistance in aggressive breast cancer

Artificial intelligence can be used to efficiently diagnose rare diseases

Artificial intelligence can be used to efficiently diagnose rare diseases

Mount Sinai joins with Paradigm and ReqMed to repurpose drug for treatment of MPS

Mount Sinai joins with Paradigm and ReqMed to repurpose drug for treatment of MPS

FDA grants Rare Pediatric Disease Designation for Immusoft’s Iduronicrin genleukocel-T

FDA grants Rare Pediatric Disease Designation for Immusoft’s Iduronicrin genleukocel-T

Discovery of molecular nets may lead to new treatment approach for heart failure

Discovery of molecular nets may lead to new treatment approach for heart failure

FDA approves drug to treat pediatric, adult patients with rare Sly syndrome

FDA approves drug to treat pediatric, adult patients with rare Sly syndrome

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

FDA grants permission to market new Seeker System for screening rare metabolic disorders in newborns

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

Researchers agree to test new stem cell gene therapy for Sanfilippo disease in human trial

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

FDA clears ArmaGen's AGT-182 IND application for treatment of Hunter syndrome

Research finding opens door to potential treatment for MPS IIIB

Research finding opens door to potential treatment for MPS IIIB

Shire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome

Shire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome

European scientists set up new therapeutic approaches to tackle gene defects

European scientists set up new therapeutic approaches to tackle gene defects

Medical researchers discover structure of potential drug target for rare genetic disease

Medical researchers discover structure of potential drug target for rare genetic disease

Researchers to use state-of-the-art brain imaging to study mucopolysaccharidosis

Researchers to use state-of-the-art brain imaging to study mucopolysaccharidosis

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

FDA grants orphan drug designation to ARMAGEN's AGT-182 for treatment of Hunter syndrome

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

Synageva BioPharma presents data on cholesteryl ester storage disease at NLA annual meeting

Synageva BioPharma presents data on cholesteryl ester storage disease at NLA annual meeting

FDA designates Synageva's sebelipase alfa as Breakthrough Therapy for early onset LAL Deficiency

FDA designates Synageva's sebelipase alfa as Breakthrough Therapy for early onset LAL Deficiency