We Need your help.

---

‘She Will Die Without This Medication’: 

Toddler’s Life Hangs In Balance After ‘Miracle’ Drug Discontinued

“We will be working with them until the bitter end, and I can’t promise that we are going to find a way to go past October, but we are going to explore every option,” 

- Dr. Elizabeth Berry-Kravis

Chicago, IL

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

The U.S. Food and Drug Administration (FDA) has a specific classification for what is known as a “rare pediatric disease.” Every condition that meets that designation is, by definition, horrible. These are the diseases that most families are blissfully unaware of, while for others the names of these diseases—Severe Combined Immunodeficiency Disease, CANDLE Syndrome, Pompe Disease—become a dark drumbeat that sounds behind each moment of their lives.

Firefly Fund co-founder Pam Andrews has a heartfelt and candid conversation with nine other NPC moms from around the world regarding recent news of the discontinuation of the medication Adrabetadex. 

This episode gives a glimpse into the difficult circumstances NPC families are facing, as well as their personal concerns and fears about their children's futures.

Episodes: Part 1 & Part 2

Experimental Treatment for Rare Children's Disease to be Discontinued

Niemann-Pick is a disease that affects memory and is sometimes referred to as Childhood Alzheimer’s, but Niemann-Pick ravages the whole body. Darsha Philips reports for NBC4 News at 4 p.m. on March 4, 2021.

Medication for a 7-year-old boy from Morrison Illinois with a rare genetic disease is being discontinued

by: Brian Gallagher

Posted: Feb 3, 2021 / 08:45 PM CST

When Drug Development for Rare Disease Hit Setback, Parents Were Stung

Families, scientists, regulators and drugmakers were all invested in development a treatment for Niemann-Pick Type C disease - so why is it taking so long?

By Amy Dockser Marcus

Photo: Pam Andrews