Amicus Therapeutics Inc

Amicus Therapeutics Inc a biotechnology company focuses on discovering developing and delivering medicines for rare diseases Its commercial product and product candidates include Galafold an oral precision medicine for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data It also develops ATGAA a novel treatment paradigm for Pompe disease enzyme replacement therapies for Pompe diseases CLN3 which is in Phase 12 clinical study to evaluate the safety and efficacy of a single intrathecal administration of an AAV serotype ATGTX502 gene therapy in patients with CLN3 and CDKL5 a gene on the Xchromosome encoding the CDKL5 protein that regulates the expression of essential proteins for normal brain development The company has collaboration and license agreements with Nationwide Childrens Hospital University of Pennsylvania and GlaxoSmithKline Amicus Therapeutics Inc was incorporated in 2002 and is headquartered in Philadelphia Pennsylvania