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.

Valley baby showing great progress with experimental Niemann-Pick Type C disease drug treatments

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

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

Parents of kids with Niemann Pick C advocate for adrabetadex

Firefly Fund is Buzzing With Activity - Firefly Fund

Jill Beirl – NNPDF

October 2023 Newsletter – NNPDF

About Us - Firefly Fund

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