Monday, July 24, 2017 4:11:45 PM
ADA-SCID at phase II/III
MPS IIIA (Sanfilippo A) about to start trials HTTP://mpssociety.org/mps/mps-iii/
HTTP://orchard-tx.com/programmes/
To be granted Rare Paediatric Disease Designation, a drug must be designed for the treatment of a serious or life-threatening disease which affects less than 200,000 patients in the United States and which primarily includes patients aged between 0 and 18 years.
This designation acknowledges that the company may qualify for a paediatric priority review voucher at the time the drug gets approved for this indication. That voucher could then be redeemed to receive a priority review of a subsequent marketing application for a different product or be transferable to other company.
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