- Amicus Therapeutics, Inc. (NASDAQ:FOLD) has strengthened its executive leadership
- It develops enzyme replacement therapies (ERTs) for lysosomal storage disorders (LSDs), including Fabry disease, Pompe disease, and Mucopolysaccharoidosis Type I (MPS I). The company also develops novel small molecules as monotherapy treatments ...
- How High Will Bluebird Bio Fly?
- Cambridge, Massachusetts-based bluebird bio (NASDAQ:BLUE) is a $710M market cap clinical-stage biotechnology company that is developing gene therapies for severe genetic and orphan diseases. Founded in 1992, the company was formerly known as ...
- Gene Therapy For Rare Genetic Disorders, Glaxo And Two Italian Groups Form
- GlaxoSmithKline, Fondazione San Raffaele and Fondazione Telethon have entered an alliance to develop gene therapies for rare genetic disorders. The therapies use stem cells taken from the patient's own bone marrow. Initially, GlaxoSmithKline (GSK) will ...
- GSK Forms Alliance for Development of Gene Therapy Techniques for Rare Diseases
- Others include beta-thalassemia, mucopolysaccharoidosis type I (MPS), globoid leukodystrophy (GLD), and chronic granulomatous disorder (CGD). All of these disorders have molecular mechanisms that are well understood and all are caused by faults in a ...
Mucopolysaccharoidosis is described in multiple online sources, as addition to our editors' articles, see section below for printable documents, Mucopolysaccharoidosis books and related discussion.
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