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.

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

BioMarin Pharmaceutical Inc. today announced an update on the GALNS Phase 1/2 extension study (MOR-100) in which patients have continued treatment on an ongoing basis.

29 Jul 2011

BioMarin commences BMN 673 Phase 1 trial in hematological malignancies

BioMarin Pharmaceutical Inc. today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.

15 Jul 2011

BioMarin to acquire Pfizer's Ireland manufacturing plant

BioMarin Pharmaceutical Inc. announced today that it has entered into a definitive agreement to acquire a bulk biologics manufacturing plant from Pfizer, located in Shanbally, Cork, Ireland.

24 Jun 2011

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

Patricia Dickson, MD, and Jennifer Yee, MD, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) researchers, will be honored on Thursday as recipients of the first UCLA Clinical and Translational Science Institute's (CTSI) Award for Translational Research in Maternal, Child, and Adolescent Health.

1 Jun 2011

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

Investigators at Nationwide Children's have received a grant from the National Institutes of Health to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.

26 May 2011

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

BioMarin Pharmaceutical Inc. announced today preliminary results from ADAPT (A Diversified Approach for PKU Treatment) in an abstract (Abstract #91, Mental Health Screening in Phenylketoniuria Clinic) presented by Barbara Burton, MD at the 2011 annual American College of Medical Genetics conference in Vancouver, Canada.

19 Mar 2011

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease

Sanfilippo disease is a rare disorder caused by the failure of enzymes to break down specific kinds of complex carbohydrates, resulting in their accumulation in cells and often severe physical and neurological problems - and sometimes early death.

1 Mar 2011

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

BioMarin Pharmaceutical announced today that it has initiated a pivotal Phase 3 trial for N-acetylgalactosamine 6-sulfatase, intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA, also called Morquio A Syndrome.

1 Feb 2011

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

BioMarin Pharmaceutical Inc. announced today that it has initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase in development for the treatment of Pompe disease.

20 Jan 2011

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

BioMarin Pharmaceutical Inc. today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK has completed review of the Clinical Trial Application (CTA) and issued a notice of acceptance for the Phase 3 trial of N-acetylgalactosamine 6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis IVA (MPS IVA). The company expects to initiate the Phase 3 pivotal trial early in the first quarter of 2011.

15 Dec 2010

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

3 Dec 2010

New study offers hope for children with MPS I genetic disorder

A new study offers hope for children born with a rare genetic disease, according to a paper published by the American Association for the Advancement of Science.

3 Dec 2010

BioMarin announces BMN-111 program for treatment of achondroplasia

BioMarin Pharmaceutical Inc. today announced its program for BMN-111, a peptide therapeutic for the treatment of achondroplasia. BioMarin plans to file an IND in the fourth quarter of 2011 and to initiate a Phase 1 clinical trial by the first quarter of 2012.

19 Oct 2010

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

BioMarin Pharmaceutical Inc. announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe disease. An investigational new drug application (IND) for BMN-701 has been submitted, investigational material has been manufactured and a Phase I/II study is expected to start in the first quarter of 2011.

30 Aug 2010

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

BioMarin Pharmaceutical Inc. announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.

18 Aug 2010

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

BioMarin Pharmaceutical Inc. announced today that the first subject has initiated treatment in a Phase 3b study (PKU-016) to evaluate the effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (PKU). BioMarin expects to report results in the first half of 2012.

18 Aug 2010

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

BioMarin Pharmaceutical Inc. announced today an update in the Phase 2 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Data reported in this announcement reflect preliminary results as of July 23, 2010. Complete top-line results are expected in the fourth quarter of 2010.

3 Aug 2010

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

BioMarin Pharmaceutical Inc. today announced financial results for the second quarter of 2010. GAAP net loss was $0.5 million ($0.01 per diluted share) for the second quarter of 2010, compared to GAAP net income of $1.3 million ($0.01 per diluted share) for the second quarter of 2009. Non-GAAP net income was $8.6 million ($0.08 per diluted share) for the second quarter of 2010, compared to non-GAAP net income of $9.0 million ($0.09 per diluted share) for the second quarter of 2009.

3 Aug 2010

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

BioMarin Pharmaceutical Inc. announced today that it has completed the Phase 1 clinical study of BMN 195, a small molecule utrophin up-regulator, for the treatment of Duchenne muscular dystrophy (DMD). The Phase 1 clinical trial was a single-center, double-blind, placebo-controlled, single-dose escalation study followed by a multiple-dose escalation study in healthy volunteers.

3 Aug 2010

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."

19 May 2010

Mucopolysaccharidosis News and Research

Further Reading

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 to acquire Pfizer's Ireland manufacturing plant

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

BioMarin announces preliminary results from ADAPT in patients suffering from PKU

Dermatan sulfate holds potential as an additional biomarker for Sanfilippo disease

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

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

New study offers hope for children with MPS I genetic disorder

BioMarin announces BMN-111 program for treatment of achondroplasia

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

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

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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