Parents of kids with Niemann Pick C advocate for adrabetadex

Parents of children with Niemann Pick Type C, NPC, hope a company is willing to manufacture adrabetadex, which they say helps their kids.

June 2021 News – NNPDF

February 2022 – NNPDF

Valley family fights for drug to save sick child

Niemann Pick C Awareness Group

Families plead with FDA for drug to treat Niemann-Pick Disease Type C

Arizona toddler with Niemann-Pick Disease finally gets 'miracle drug

PDF) International consensus on clinical severity scale use in evaluating Niemann–Pick disease Type C in paediatric and adult patients: results from a Delphi Study

October 2023 Newsletter – NNPDF

Fundraiser by Sara McGlocklin and NPC Mamas : Save Our Medicine

January – February 2022 Newsletter – NNPDF

APMRF Fall Newsletter by parseghianfund - Issuu

Mallinckrodt pulls out of cyclodextrin trial – NPC community will continue to fight for it.

A terrible disease, children at risk, and a promising treatment that's about to vanish: Part II