Orphan Drug designation for Retinitis Pigmentosa treatment

 GenSight’s developing a drug GS030,based on optogenetics, a technology that makes cells responsive to light to treat Retinitis Pigmentosa (RP).
RP is a rare disease that can be occur in isolation or as part of a syndrome.
Some forms of CDG present with RP.
​ #CDG #OrphanDrug