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Latest Inborn errors of metabolism Stories

2009-06-02 07:00:00

- Company preparing to initiate phase II trial in hepatic encephalopathy - SOUTH SAN FRANCISCO, Calif., June 2 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced top-line results from a phase I study of HPN-100 in patients with liver cirrhosis.

2009-05-29 08:03:25

A dog born with a deadly disease that prevents the body from using stored sugar has survived 20 months and is still healthy after receiving gene therapy at the University of Florida — putting scientists a step closer to finding a cure for the disorder in children.

2009-05-15 08:00:00

National Organization for Rare Disorders (NORD) Recognizes BioMarin's Efforts to Expand Treatment Options for Patients with PKU NOVATO, Calif., May 15 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.

2009-05-05 07:00:00

- Company preparing to initiate phase III clinical trial - SOUTH SAN FRANCISCO, Calif., May 5 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced that HPN-100 has received orphan product designation from the U.S.

2009-04-21 15:00:00

NOVATO, Calif., April 21 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.

2009-04-06 04:24:00

LEIDEN, The Netherlands, April 6 /PRNewswire/ -- The Dutch Orphan Disease Registry Consortium is set up in order to build a disease registry framework to collect information on rare diseases, encourage drug development and optimize patient care.

2009-03-30 07:00:00

- Company preparing to initiate phase III trial - SOUTH SAN FRANCISCO, Calif., March 30 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced top-line results from their phase II study of HPN-100 for the treatment of urea cycle disorders.

2009-03-18 08:00:00

NOVATO, Calif., March 18 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.

2009-02-18 15:00:00

CRANBURY, N.J., Feb. 18 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) announced today that Stephen Bloch, M.D., General Partner with Canaan Equity Partners, has resigned from the Company's Board of Directors effective February 17, 2009. Dr.


Word of the Day
shotta
  • An armed gangster.
The word 'shotta' is an alteration of 'shooter'.