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Latest Glycogen storage disease type II Stories

2008-10-17 15:00:23

Genzyme Corporation (Nasdaq: GENZ) today announced that its U.S. Biologic License Application (BLA) for Myozyme(R) (alglucosidase alfa) produced at the 2000 L bioreactor scale will be discussed this Tuesday, October 21st, at a public meeting of the FDA's Endocrinologic and Metabolic Drugs Advisory Committee in Silver Spring, Maryland. This product is intended for the treatment of Pompe disease, a progressive, debilitating and life-threatening inherited disorder affecting approximately 2,000...

2008-09-22 09:00:12

MONTREAL, Sept. 22 /PRNewswire/ -- Enobia Pharma, a biotech company focused on developing novel therapeutics for serious bone disorders, today announced key appointments to add commercial and manufacturing expertise to the Company's senior management team in conjunction with the recent initiation of two clinical trials of ENB-0040 for the treatment of hypophosphatasia. Julie Anne Smith has been appointed as Enobia's Vice President, Chief Commercial Officer, and Jayant Aphale, PhD., MBA has...

2008-08-07 18:01:54

CRANBURY, N.J., Aug. 7 /PRNewswire-FirstCall/ -- Amicus Therapeutics , a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced financial results for the second quarter of 2008. On a reported basis calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Amicus announced a net loss attributable to common stockholders of $0.41 per share ($0.33 per share on a...

2008-08-04 09:01:05

CRANBURY, N.J., Aug. 4 /PRNewswire-FirstCall/ -- Amicus Therapeutics , a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, announced today that it has successfully completed an End of Phase 2 meeting for Amigal(TM) with the U.S. Food and Drug Administration (FDA). The FDA indicated that the data from the completed Phase 2 clinical studies of Amigal support the start of Phase 3 development and...

2008-07-31 09:01:26

CRANBURY, N.J., July 31 /PRNewswire-FirstCall/ -- Amicus Therapeutics , a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, will announce second quarter 2008 financial results after the regular close of markets on Thursday, August 7, 2008. The results announcement will be followed by a live conference call and webcast that afternoon at 5 p.m. EDT. Interested participants and investors may access...

2006-04-28 16:40:00

By Lisa Richwine WASHINGTON (Reuters) - The first drug to treat a rare, debilitating muscle disease that can kill infants within a year won U.S. approval on Friday, health officials said. Genzyme Corp. developed the drug, Myozyme, to treat Pompe disease, which afflicts fewer than 10,000 people worldwide. It causes severe muscle weakness and breathing trouble that can be fatal, but the progression can vary. If the disease emerges in infancy, it usually causes an enlarged heart and kills within...


Word of the Day
grass-comber
  • A landsman who is making his first voyage at sea; a novice who enters naval service from rural life.
According to the OED, a grass-comber is also 'a sailor's term for one who has been a farm-labourer.'