All IndicationsNeurology & CNS
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Pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A
1
Companies
1
Drug Programs
0
Phase 3
0
Upcoming PDUFAs
Competitors
| Company | Drug | Phase | Designations | Timeline | Trial ID |
|---|---|---|---|---|---|
| RETA Reata Pharmaceuticals | rebisufligene etisparvovec-hopf | Approved | — | — | — |
Development Stage Distribution
Approved
1
PDUFA Timeline
No upcoming PDUFA dates for this indication.
Not financial advice
Signals are probabilistic and based on historical data — past performance doesn't guarantee future results. Do your own research.