Latest Inborn errors of metabolism Stories
Imagine being forced to say no to a child crying for more food at supper. Sadly, Margie Fischer doesn't have to imagine it; that was normal life at her family's dinner table for years.
Company Expects to Deliver Multiple Clinical Milestones throughout the Year; Amigal to Initiate Phase 3 Global Development in Second Quarter; Continued Strong Financial Position CRANBURY, N.J., Jan.
The life of a Minnesota boy suffering with a rare and serious form of the genetic disorder Pompe disease has been extended after treatment with a drug typically used to suppress the immune system of those with cancer and rheumatoid arthritis.
CRANBURY, N.J., Jan. 8 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced the election of Sol J. Barer, Ph.D., to its Board of Directors. Dr. Barer currently serves as Chairman and Chief Executive Officer of Celgene Corporation. As an industry veteran, Dr.
NOVATO, Calif., Dec. 9 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
SOUTH SAN FRANCISCO, Calif., Nov. 12 /PRNewswire/ -- Hyperion Therapeutics, Inc.
NOVATO, Calif., Nov. 6 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
NOVATO, Calif, Nov. 3 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today the initiation of the Morquio Clinical Assessment Program (MorCAP) for patients with the lysosomal storage disease Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome.
By HARWARD, Esther WHEN TWO of Tauranga mum Jenny Noble's three children were diagnosed with a crippling bone disease, they told her they were in so much pain they wanted to die.
NOVATO, Calif., Sept. 25 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.