June 6, 2017
Amicus Therapeutics Receives Rare Pediatric Disease Designation for SD-101 for Patients with Epidermolysis Bullosa
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March 27, 2017
Jaipur Doctor to be Part of Drafting Policy on Treating Rare Disease
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March 12, 2016
Pompe Diagnosis Brings Relief After One Woman’s Long Search
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January 26, 2013
BioMarin Seeks to Test Enzyme-Replacement Therapy in Batten Disease Patients
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May 16, 2012
Pfizer Announces FDA Approval of ELELYSO™ for Type 1 Gaucher Disease and Personal Support Program For Patients
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May 13, 2010
Niemann-Pick Disease: An Update on Joseph Colton
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March 4, 2009
Cherry Red Spot – Telltale Sign of Neurometabolic Disorders
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