ArticleMolecular genetics and metabolism reports2023

Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.

Reena Sharma et al.PubMed ↗Full text ↗Publisher ↗

No numbers read from the abstract.