February 27, 2018
Generation Bio Raises $100 Million to Advance Re-Dosable Gene Therapy
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January 7, 2017
Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-201 Gene&hel...
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September 6, 2012
Our Faith Continues To Grow Despite Methacromatic Leukodystrophy (MLD) Diagnosis
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August 22, 2012
Fatal Sanfilippo Syndrome Strikes Three Children in Same Family
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July 1, 2012
Six Year Old with Juvenile Metachromatic Leukodystrophy Is Mom’s Super Hero
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October 27, 2010
Former BioMarin exec targets orphan drugs
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