Browsing Tag
Niemann-Pick Disease Type C
3 posts
New hope for Niemann-Pick? Mandos Health drug shows survival benefit ahead of ANA 2025
Mandos Health to present new data on adrabetadex for Niemann-Pick Disease Type C at ANA 2025. Find out why survival and biomarker data matter now.
IntraBio’s AQNEURSA earns positive CHMP opinion, bringing Niemann-Pick disease type C therapy closer to EU approval
IntraBio’s AQNEURSA earns positive CHMP opinion for Niemann-Pick disease type C, moving closer to EU approval after strong Phase III data and U.S. FDA nod.
FDA committee endorses Zevra Therapeutics’ arimoclomol as a treatment for Niemann-Pick Disease Type C
In a notable advancement for rare disease treatment, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) has received a significant endorsement…