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Latest Glycogen storage disease type II Stories

2011-03-08 06:00:00

CRANBURY, N.J., March 8, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease. Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase...

2011-02-16 07:30:00

CRANBURY, N.J., Feb. 16, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced that additional positive data from the ongoing Phase 2 extension study of its investigational drug Amigal(TM) (migalastat HCl) for Fabry disease will be presented at the Lysosomal Disease Network WORLD Symposium in Las Vegas, Nevada, February 16-18th, 2011. In addition, the Company announced that it will present encouraging data from its preclinical studies evaluating the co-administration of...

2011-02-14 15:05:00

CRANBURY, N.J., Feb. 14, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the fourth quarter and full year 2010. The Company also provided a review of planned key milestones for 2011, which includes the announcement of top line results from the ongoing Phase 3 Study of Amigal(TM) (migalastat HCl) for Fabry Disease. Fourth Quarter and Full-Year 2010 Financials Summary Amicus announced net loss attributable to common stockholders of $0.48 per...

2011-01-19 08:00:00

NOVATO, Calif., Jan. 19, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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 (IGF2-GAA) in development for the treatment of Pompe disease. "We have a strong track record of quickly developing enzyme replacement therapies for unmet medical needs and expect to leverage our clinical and regulatory experience and manufacturing...

2011-01-11 07:00:00

CRANBURY, N.J., Jan. 11, 2011 /PRNewswire/ -- Amicus Therapeutics (Nasdaq: FOLD) today will provide the Company's business outlook and expected key milestones for 2011 at the 29th Annual J.P. Morgan Healthcare Conference. At the conference, Amicus is providing an update on its three key areas of focus: Amigal (migalastat hydrochloride) for the treatment of Fabry Disease, the evaluation of pharmacological chaperones co-administered with ERT, and the investigation of pharmacological...

2010-12-02 00:52:40

In a study that supports the need for newborn screening and early treatment for a rare genetic disorder, researchers at Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and Iowa State University found enzyme replacement therapy beginning at birth eliminated almost all of the symptoms associated with mucopolysaccharidosis type I, or MPS I, in animal models. The study, which was published today in the journal, Science Translational Medicine, is the first to...

2010-11-08 15:01:00

CRANBURY, N.J., Nov. 8, 2010 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the quarter ended September 30, 2010, provided an update on its product development pipeline, including its Phase 3 program with Amigal (migalastat HCl) for the treatment of Fabry disease and reaffirmed the financial strength of the Company. John F. Crowley, Chairman and CEO of Amicus Therapeutics stated, "During the third quarter we made significant progress...

2010-08-30 08:00:00

NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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...

2010-08-17 15:27:00

NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) 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. Under the terms of the...

2010-08-05 15:00:00

CRANBURY, N.J., Aug. 5 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the quarter ended June 30, 2010, provided an update on its product development pipeline and reiterated guidance for timelines related to its Phase 3 program with Amigal (migalastat HCl) for the treatment of Fabry disease. John F. Crowley, Chairman and CEO of Amicus Therapeutics stated, "Our global Phase 3 program with Amigal for Fabry disease is our number one...