International Niemann–Pick Disease Alliance
| Disease | Drug | Route | Sponsor | Locations |
|---|---|---|---|---|
| Niemann-Pick C | Arimoclomol | oral | Zevra | United States, some European countries |
| Niemann-Pick C | Adrabetadex | intrathecal | Mandos | United States only |
Further resources to understand clinical trials and drug development:
What patients need to know about clinical trials – resource from the FDA https://www.fda.gov/patients/clinical-trials-what-patients-need-know/basics-about-clinical-trials
The drug development process – resource from the FDA https://www.fda.gov/patients/learn-about-drug-and-device-approvals/drug-development-process