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.
BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

BioMarin commences GALNS Phase 3 trial in patients with Morquio A Syndrome

Biomarin commences BMN 701 Phase 1/2 trial for treatment of Pompe disease

Biomarin commences BMN 701 Phase 1/2 trial for treatment of Pompe disease

MHRA issues notice of acceptance for BioMarin's GALNS Phase 3 trial in MPS IVA

MHRA issues notice of acceptance for BioMarin's GALNS Phase 3 trial in MPS IVA

MHRA issues notice of acceptance for BioMarin's BMN 673 for genetically-defined cancers

MHRA issues notice of acceptance for BioMarin's BMN 673 for genetically-defined cancers

New study offers hope for children with MPS I genetic disorder

New study offers hope for children with MPS I genetic disorder

BioMarin announces BMN-111 program for treatment of achondroplasia

BioMarin announces BMN-111 program for treatment of achondroplasia

BioMarin's BMN-701 receives FDA orphan drug designation for treatment of Pompe disease

BioMarin's BMN-701 receives FDA orphan drug designation for treatment of Pompe disease

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin commences Phase 3b study of Kuvan's neuropsychiatric effects in phenylketonuria patients

BioMarin commences Phase 3b study of Kuvan's neuropsychiatric effects in phenylketonuria patients

BioMarin provides update on PEG-PAL Phase 2 clinical study for phenylketonuria

BioMarin provides update on PEG-PAL Phase 2 clinical study for phenylketonuria

BioMarin reports $0.5 million GAAP net loss for second-quarter 2010

BioMarin reports $0.5 million GAAP net loss for second-quarter 2010

BioMarin completes BMN 195 Phase 1 clinical trial for Duchenne muscular dystrophy

BioMarin completes BMN 195 Phase 1 clinical trial for Duchenne muscular dystrophy

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

BioMarin Pharmaceutical reports GAAP net income of $4.7M for fourth-quarter 2009

BioMarin Pharmaceutical reports GAAP net income of $4.7M for fourth-quarter 2009

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

BioMarin announces update on BMN 110 Phase I/II trial for Morquio A Syndrome

Naglazyme approved in Japan

Naglazyme approved in Japan

One injection of gene therapy spreads through brain in animal study

One injection of gene therapy spreads through brain in animal study

Genzyme receives Japanese marketing approval for Elaprase

Genzyme receives Japanese marketing approval for Elaprase

First treatment for Hunter disease

First treatment for Hunter disease

FDA approves Elaprase (idursulfase) for Hunter syndrome

FDA approves Elaprase (idursulfase) for Hunter syndrome