Wolman Disease
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Clinical Development Program in LAL Deficiency | RARE Project
crdnetwork.org — “LAL Deficiency, a lysosomal storage disorder, has early and late onset phenotypes, also referred to as Wolman Disease or Cholesteryl Ester Storage Disease” View full resource at crdnetwork.org
Most Recently Shared on November 22, 2011 at 12:07 am By:
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Clinical Development Program in LAL Deficiency http://t.co/ROweuHwi (via @RAREproject)
Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy
news-medical.net — “Synageva BioPharma Corp., a privately held biopharmaceutical company, today announced its filing with the U.S. Food and Drug Administration (FDA) requesting orphan drug designation for its program, SBC-102. This program is an enzyme replacement therapy in preclinical development to treat lysosomal acid lipase (LAL) deficiency, also known as Wolman disease or cholesteryl ester storage disease (CESD), a condition for which there is currently no effective treatment.” View full resource at news-medical.net
Most Recently Shared on May 4, 2010 at 1:36 pm By:
Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy: Synageva BioPharma Co... http://bit.ly/c0taPL
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