Cyclo Therapeutics has shared topline results from its Phase 3 TransportNPC study, which included Australian participants. While the study’s main endpoint was not statistically significant, important findings—including a reported survival benefit for children with infantile-onset NPC—warrant close attention and further review.

Watch the Royal Children’s Hospital Grand Rounds presentation exploring Australia’s first Standard of Care for Niemann–Pick disease type C.

Frank W. Pfrieger, PhD Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD)...

Summaries of latest research advances related to Niemann-Pick diseases, acid sphingomyelinase deficiency (ASMD) and Niemann-Pick type C...