FIGHTING FOR THEIR LIVES

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Right now, it is crucial to amplify the discussion around NPC, those who are affected, and why the FDA’s denial of arimoclomol could present immense issues for patients.

Moving forward, the fight for NPC research and treatments is on the horizon. The entire NPC community is going through a frightening time, with both potential treatments currently halted by the FDA.

Mandos, LLC Agrees to Acquire Adrabetadex from Mallinckrodt Pharmaceuticals

“We look forward to engaging with this passionate community of patients, caregivers, clinicians, and regulators over the coming months, once the transaction is approved. We care deeply about ensuring access for patients and are committed to supporting efforts to improve the lives of people with NPC1,” said Scott Riccio, EVP, Patient & Community Engagement at Mandos.

Mallinckrodt Sells Rare Disease Drug

‘There’s so much uncertainty’: As Mallinckrodt sells rare disease drug, parents worry about access

After at least two possible buyers did not pan out, Mallinckrodt reached a deal on Wednesday to sell the drug to a little-known company called Mandos for $1 million and $2 million more in milestone payments, according to court documents. And Mallinckrodt agreed to secure enough supply to allow access to the drug for potentially up to two years under expanded access.

Woodrow Miller, a 21-month-old toddler, has now received his first infusion of Adrabetadex, an experimental medication for Niemann-Pick Type C disease.

ABC15 has been in touch with Woodrow's family every day since they left for Chicago's Rush University Medical Center last week, to start Woodrow's treatment. His family tells us Woodrow took his first spinal infusion like a champ.

The experimental medication gives hope to so many families who have children suffering from Niemann-Pick Type C, a rare and fatal disease that eventually takes away a child's ability to walk, talk, swallow, and breathe. It has been likened to a childhood form of Alzheimer's.

'We know it works': Families & Doctors fight move to discontinue medication

Imagine being a parent and being told your child is going to die. That's the reality for an Omaha couple who says recent news about their son's disease feels like a nightmare starting all over again.

For the parents of a 5-year-old, the news is devastating.

They recently received word that the company that makes their son's medicine is taking it away.

‘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