Skip to main content

Minnesota toddler's life hangs in balance as drug for her rare disease is set to be discontinued

"Our kids will die without their medicine," says a fundraiser started by NPC moms.
Niemann-Pick Type C

The cerebellum of a mouse brain affected by Niemann-Pick Type C (NPC), a rare neurological disorder. NIH researchers are conducting studies to advance treatments for NPC and similar rare diseases.

The family of a Minnesota toddler with a deadly disease is fighting for her life after a drugmaker discontinued a medicine that has helped treat her condition. 

Sara and Mitch Peterka, of Minnetonka, are among a group of parents around the world doing all they can to spread the urgent message that they hope will save the lives of children with Niemann-Pick Type C (NPC), a rare neurological disorder.

Emma Peterka, 2, has NPC, and her parents fear she'll die if Mallinckrodt, the pharmaceutical company that makes a drug that treats the disease, discontinues production of adrabetadex as is planned later this year. 

"Without this medicine, they will needlessly suffer and die - losing their ability to speak, walk, talk, and more. This is literally life or death and they are all about to lose access to this medicine in only a few months," explains an online fundraiser for NPC families

Mitch Peterka told WCCO-TV that Emma "will die without this medication," while Sara said that news of the drug being discontinued was "devastating." 

Mallinckrodt plans to stop providing adrabetadex to eligible NPC patients in October 2021. The drugmaker issued a statement saying that although some children have benefitted from adrabetadex, clinical studies show there is not enough evidence to confirm the medicine helps treat NPC.   

"We are aware that some families and their physicians report seeing benefit in children being treated with adrabetadex. These reports and case studies were the reason we were hopeful the scientific study would also demonstrate evidence of benefit for patients with NPC. However, after exhaustive analysis of clinical data, there is no clear evidence that adrabetadex changes or improves disease progression across the group of patients that were studied."

The online fundraiser was launched by a group of NPC moms, who liken the condition to a combination of Alzheimers, Parkinson's and ALS. 

It has raised more than $122,000 as of this writing, and all proceeds will go to a charitable organization that are used to "assemble an expert team to champion our children's benefit and need to safeguard access to this medicine." 

According to Mayo Clinic, NPC mainly affects children, has "no known cure and is sometimes fatal." The disease causes a progressive loss of nerve, brain and other organ function. 

Next Up

cats

Reward to find person who left kittens to die in freezing WI cold

The cats were thrown from a vehicle, according to a witness.

metro mobility bus

Charges: Man led police on multi-city pursuit in stolen Metro Mobility bus

The bus had been left unattended with the keys inside when it was stolen.

Bell Ramsey Co. Jan 22 - crop

Charges: Man fatally shot mother of his child during argument

Officers found her on the ground outside the couple's home.

covid

Minnesota's COVID-19 update for Wednesday, January 26

More than 15,000 new cases in today's update.

Kwesi Adofo-Mensah

The biggest personnel decisions facing the new GM

Kwesi Adofo-Mensah has some tricky personnel decisions coming up in the near future

Kwesi Adofo-Mensah

What to know about new Vikings GM Kwesi Adofo-Mensah

The former Wall Street trader is an analytics guru who appreciates collaboration.

metro transit southwest light rail green line extension construction

Southwest LRT may cost $210M more, take 3 years longer to build

The Met Council is proposing a settlement with one of its contractors due to disputes over construction changes.

Rick Spielman

Spielman: 'That head coach has to have a relationship with that QB'

"I think the No. 1 thing you’re looking for is that head coach has to have a relationship with that quarterback."

Screen Shot 2022-01-26 at 6.36.25 AM

Gallery: France 44 to build two-story addition, rooftop patio

The Minneapolis liquor store and cheese shop is stepping up its events and class offerings.

Related

Screen Shot 2021-05-25 at 9.09.14 PM

Minnesota boy diagnosed with rare tick-borne disease

The boy is believed to have contracted the disease from a tick in the Buffalo area.

heart monitor, hospital

25 cases of rare child disease possibly linked to COVID-19 in MN

It's extremely rare, but health officials want parents to be aware.

Screen Shot 2021-10-13 at 3.17.46 PM

Minnesota family learns their baby has extremely rare, fatal disease

The baby's mother is hoping to provide her son with the best life possible.

Rare neurological disease reported in six Minnesota kids

Officials are investigating this "rare but potentially severe" condition.

coronavirus

MN officials watching for child syndrome linked to COVID-19, but say it's 'rare'

Health experts in Minnesota are keeping an eye out for the rare syndrome.

7th MN child diagnosed with disease that attacks nervous system

The Centers for Disease Control and Prevention is investigating.