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Latest Inborn errors of metabolism Stories

2009-10-21 06:04:00

CAMBRIDGE, Massachusetts, October 21 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces plans to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease, by the end of the year. The Company also announces that a treatment protocol for REPLAGAL, filed at the request of FDA, has been approved, and that...

2009-10-14 07:30:00

MILWAUKEE, Oct. 14 /PRNewswire/ -- ZyStor Therapeutics, Inc., a biotechnology company developing a new class of targeted protein therapeutics for the treatment of Lysosomal Storage Diseases using the Company's proprietary Glycosylation Independent Lysosomal Targeting technology, today announced that it has received clearance from the U. S. Food and Drug Administration to proceed into a clinical trial for its first drug candidate, ZC-701, an enzyme replacement therapy for the treatment of...

2009-08-19 08:00:00

NOVATO, Calif., Aug. 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that data from clinical studies of Kuvan(R) (sapropterin dihydrochloride) and Naglazyme(R) (galsulfase) will be presented at the 11th Annual Meeting of the International Congress of Inborn Errors of Metabolism (ICIEM) in San Diego, California, August 29 through September 2, 2009. "We look forward to a number of abstracts, posters and platform presentations that will highlight a...

2009-08-13 10:44:37

 Supplementing obese rats with the nutrient carnitine helps the animals to clear the extra sugar in their blood, something they had trouble doing on their own, researchers at Duke University Medical Center report.A team led by Deborah Muoio (Moo-ee-oo), Ph.D., of the Duke Sarah W. Stedman Nutrition and Metabolism Center, also performed tests on human muscle cells that showed supplementing with carnitine might help older people with prediabetes, diabetes, and other disorders that make...

2009-08-05 09:55:00

Researchers from CIC bioGUNE's Structural Biology Unit and Columbia University (New York) have conducted a joint research project, published in the prestigious scientific journal Structure, to gain in-depth knowledge of the structure of pyruvate carboxylase when it is in solution (in the "natural" state).Pyruvate carboxylase is a metabolic enzyme that plays a fundamental role in the metabolism of fatty acids (the components of fats) and sugars. When its function is not adequately performed...

2009-07-16 01:00:00

LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Type I Gaucher disease. Shire is working with the FDA to determine subsequent steps and timing for the filing of its NDA. Fast Track designation is an FDA...

2009-07-13 08:00:00

NOVATO, Calif., July 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that patient enrollment has been completed for the Phase I/II clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010. "The efficient enrollment of twenty patients...

2009-07-06 14:01:00

LEXINGTON, Massachusetts, July 6 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces that, at the request of the FDA, in view of a potential restriction on the availability of the current approved and marketed treatment for Gaucher Disease patients, it has filed a treatment protocol for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher Disease. If approved by the FDA, the...

2009-06-30 07:00:00

SOUTH SAN FRANCISCO, Calif., June 30 /PRNewswire/ -- Hyperion Therapeutics, a privately held specialty pharmaceutical company focused on the development of therapies that address critical unmet needs in the areas of gastroenterology and hepatology, today announced it has closed a $60 million Series C financing. New investors Bay City Capital and Panorama Capital co-led the financing. Series A and B investors Highland Capital Partners, NEA, and Sofinnova Ventures also participated. (Logo:...

2009-06-22 07:00:00

- FDA Agreement Reached on Phase 3 Study Design, Primary Endpoint and Histological Methodology - CRANBURY, N.J., June 22 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced it has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the U.S. Food and Drug Administration (FDA) on the key protocol design elements of the pivotal...


Word of the Day
tessitura
  • The prevailing range of a vocal or instrumental part, within which most of the tones lie.
This word is Italian in origin and comes from the Latin 'textura,' web, structure.