 |
adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA |
|---|---|
| Trade Name | |
| Orphan Indication | Late Infantile Neuronal Ceroid Lipofuscinosis |
| USA Market Approval | USA |
| USA Designation Date | 2016-03-16 00:00:00 |
| Sponsor | Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 |
Related Access Program