Our children are fighting a progressive and fatal disease called
Niemann-Pick Type C (NPC).
The medication that allows them to live is being taken away in October 2021.
Imagine your child gives their everything to live, laugh, and grow. They have an aggressive and relentless fatal disease, think childhood Alzheimer's crossed with Parkinson's, crossed with ALS, but in young children.
It's called Niemann-Pick Type C (NPC).
They are living and thriving because of a miraculous, life-saving medication. It's called Abdrabetadex...
...then one day, you suddenly learn that it's going to be ripped away in a matter of months.
Many children on this drug were not in the clinical trial that was “inconclusive”. Even though this medicine keeps our kids alive.
Without this medicine, our children's near future will likely be falls, tears, loss of skills, choking, seizures, weakness, loss of speech, motion, and independence
Mallinckrodt Pharmaceuticals is taking it away in October 2021 based on the results of a 1 year trial study. Johnson & Johnson also makes and can supply this medicine, but right now we can’t access it.
Without Abdrabetadex, there is nothing to fight back the fatal disease. A disease that will steal everything, including life itself.
‘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
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.
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