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.
Renaissance in drug development for rare diseases

Renaissance in drug development for rare diseases

Synageva BioPharma reports study results of sebelipase alfa in adults with late onset LAL Deficiency

Synageva BioPharma reports study results of sebelipase alfa in adults with late onset LAL Deficiency

Researchers to develop new diagnostic tools and treatments for people with rare diseases

Researchers to develop new diagnostic tools and treatments for people with rare diseases

ArmaGen closes $17M Series A financing

ArmaGen closes $17M Series A financing

Greenhouse-grown corn seeds could help treat lysosomal storage disease

Greenhouse-grown corn seeds could help treat lysosomal storage disease

Researchers discover genetic cause of type VII mucopolysaccharidosis in Brazilian Terriers

Researchers discover genetic cause of type VII mucopolysaccharidosis in Brazilian Terriers

CHOC Children's research project receives $5.5M CIRM grant

CHOC Children's research project receives $5.5M CIRM grant

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

EMA grants orphan designations to Ultragenyx UX001, UX003 for treatment of HIBM and MPS 7

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

FDA grants orphan drug designation to Ultragenyx UX003 for treatment of MPS 7

GSK, Angiochem to develop and commercialize LSD treatments

GSK, Angiochem to develop and commercialize LSD treatments

BioMarin to initiate BMN-111 Phase 1 trial in achondroplasia

BioMarin to initiate BMN-111 Phase 1 trial in achondroplasia

EMA CHMP recommends approval for BioMarin's Novato manufacturing facility expansion

EMA CHMP recommends approval for BioMarin's Novato manufacturing facility expansion

BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

BioMarin commences GALNS Phase 2 study in mucopolysaccharidosis IVA

Positive data from BioMarin's MOR-100 Phase 1/2 extension study on Morquio syndrome

Positive data from BioMarin's MOR-100 Phase 1/2 extension study on Morquio syndrome

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin to acquire Pfizer's Ireland manufacturing plant

BioMarin to acquire Pfizer's Ireland manufacturing plant

Researchers receive CTSI Award for Translational Research in Maternal, Child, and Adolescent Health.

Researchers receive CTSI Award for Translational Research in Maternal, Child, and Adolescent Health.

Investigators receive NIH grant to help move gene therapy for MPS IIIB toward clinical trials

Investigators receive NIH grant to help move gene therapy for MPS IIIB toward clinical trials

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease