This is the second Blog Post in a series over the next week that will examine Lysosomal Storage Disorders (LSDs) in the rare disease and orphan drug space. This Blog Post presents an advocacy group that receives two FDA Orphan Drug Designations (ODDs) in 2013 for treatment of rare diseases. The chart below identifies the gene therapy that receives FDA ODD where the sponsor is a rare disease advocacy group.
|recombinant adeno- associated virus vector AAV2/rh8 expressing human B-hexosaminidase A and B subunits||03-25-2013||Sandhoff Disease|
|recombinant adenovirus vector AAV2/rh8 expressing human B-hexosaminidase A & B subunits||03-25-2013||Tay-Sachs Disease|
** “Generic Name” Column Link = Is the FDA Orphan Drug Product Designation Database Record.
The National Tay-Sachs and Allied Diseases Association (NTSAD) announces in June 2013 that the FDA…
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