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development pipeline
Pipeline – Drugs for rare diseases
| NAME |
ORIGINATOR |
INDICATION |
DEVELOPMENT STATUS |
| Carbaglu® |
Orphan Europe (Recordati) |
Organic acidemias |
Approved in the EU, Phase III in U.S.A. |
| Cystadrops® |
Orphan Europe
(Recordati) |
Ocular cystinosis |
Phase II |
|