dm+d

Unassigned

New Medicines

Niemann-Pick disease, type C (NPC) in adults and children

Information

New molecular entity
Mallinckrodt
Mallinckrodt

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

A unique form of cyclodextrin.
Niemann-Pick disease, type C affects approximately 0.1 in 10,000 people in the European Union [3].
Niemann-Pick disease, type C (NPC) in adults and children
Intrathecal

Further information

Yes
To be confirmed