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NOVATO, Calif., Feb. 1, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
NOVATO, Calif., Jan. 19, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc.
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.
Researchers believe that newborn screening and early treatment for a rare genetic disorder, mucopolysaccharidosis type I (MPS I), is needed so that enzyme replacement therapy can begin at birth and eliminate all the symptoms associated with MPS I.
A new study offers hope for children born with a rare genetic disease.
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.
SHENYANG, China, Nov. 29, 2010 /PRNewswire-Asia-FirstCall/ -- 3SBio Inc.
SOUTH PLAINFIELD, N.J., Oct. 26 /PRNewswire/ -- PTC Therapeutics, Inc. today announced that it is expanding the development of ataluren, an investigational new drug, with the initiation of a Phase 2 clinical trial in nonsense mutation methylmalonic acidemia (nmMMA).
NOVATO, Calif., Sept. 1 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
- Withering but not falling off, as a blossom that persists on a twig after flowering.