Mucopolysaccharidoses News and Research

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Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Cascade aggravates hereditary storage diseases

Cascade aggravates hereditary storage diseases

New therapeutic approach may delay neurodegeneration in rare genetic disease

New therapeutic approach may delay neurodegeneration in rare genetic disease

Gene editing within a living person for the first time

Gene editing within a living person for the first time

Newly discovered biomarker linked to rare metabolic disorders may facilitate better diagnosis

Newly discovered biomarker linked to rare metabolic disorders may facilitate better diagnosis

Synageva announces publication of LAL Deficiency review in Atherosclerosis

Synageva announces publication of LAL Deficiency review in Atherosclerosis

LA BioMed researchers test investigational drug for treatment of Sanfilippo B syndrome

LA BioMed researchers test investigational drug for treatment of Sanfilippo B syndrome

Genzyme marks 2013 International Rare Disease Day

Genzyme marks 2013 International Rare Disease Day

SLU researchers to test new screening method for MPS disorders among infants

SLU researchers to test new screening method for MPS disorders among infants

Researchers discover genetic cause of type VII mucopolysaccharidosis in Brazilian Terriers

Researchers discover genetic cause of type VII mucopolysaccharidosis in Brazilian Terriers

Scientists develop simple, reliable test to identify biomarkers for MPS

Scientists develop simple, reliable test to identify biomarkers for MPS

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease

Study suggests a new improved treatment option for children with Hurler's syndrome

Study suggests a new improved treatment option for children with Hurler's syndrome

Contribution of glycosaminoglycans to skeletal abnormalities in patients with lysosomal storage diseases demonstrated

Contribution of glycosaminoglycans to skeletal abnormalities in patients with lysosomal storage diseases demonstrated

New understanding of Sly Syndrome

New understanding of Sly Syndrome