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.

Hope For Marian - Our metabolic geneticist was on leave since January when all this went down. He just came back, walked into our appointment and said, “we cannot lose this treatment.

About Us - Firefly Fund

Fundraiser by Sara McGlocklin and NPC Mamas : Save Our Medicine

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

FDA needs to be more flexible assessing rare disease treatments - STAT

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

She Will Die Without This Medication': Toddler's Life Hangs In Balance After 'Miracle' Drug Discontinued - CBS Minnesota

October 2023 Newsletter – NNPDF

APMRF Fall Newsletter by parseghianfund - Issuu

September 2020 – NNPDF

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

Community News – NNPDF

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

January 2021 – NNPDF

Dr. Oz Show features “Baby Alzheimer's”; Niemann-Pick disease parents, types A and C