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.

Fundraiser by Sara McGlocklin and NPC Mamas : Save Our Medicine

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

June 2021 News – NNPDF

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.

January 2021 – NNPDF

Save Our Medicine:” Fighting for NPC Med Access pt. 3 - Patient Worthy

Parents of kids with Niemann Pick C advocate for adrabetadex

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

Experimental Treatment for Rare Children's Disease to be Discontinued – NBC Los Angeles

Jill Beirl – NNPDF

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