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Abby and Family Relieved after FDA Approves Two Treatments for Rare Disease

This video and article originally appeared on 12onyourside.com. Visit their website to learn more.


CHESTERFIELD, Va. (WWBT) – At PARK365, you can find Abby Alvey exploring and showing her love for all things pink and purple.

9-year-old Abby Alvey
9-year-old Abby Alvey(Source;WWBT | WWBT)

One thing the 9-year-old Chesterfield girl has shown her parents, Garland and Melissa, is resilience and strength during her journey with childhood dementia.

“She‘s really not just changed our lives; she’s changed everyone’s life around her,” said Garland Alvey, Abby’s father.

9-year-old Abby Alvey walks with her parents, Garland and Melissa.
9-year-old Abby Alvey walks with her parents, Garland and Melissa.(Source;WWBT | WWBT)

Abby was diagnosed with Niemann–Pick Disease Type C, a rare neurodegenerative disorder that can slowly take away all your abilities.

“It definitely rocked all of our worlds,” said Abby‘s father, Garland. “We thought we were in this rollercoaster of emotions thinking that we figured it out and she‘s fine and realizing that she wasn’t.”

The 9-year-old started a clinical trial for Adrabetadex, a medication Abby’s family said to put a stop to her symptom progression.

Abby Alvey
Abby Alvey(Source; Abby Strong Fights NPC | Abby Strong Fights NPC)

“It’s been a miracle,” said Garland Alvey. “She’s going to school every day.”

Abby’s family has been fighting to expand access to one of the several medications through FDA approval.

“There was a point there we felt really helpless that we would never get any of these four or five treatments across the finish line,” said Garland Alvey.

This journey reached a major milestone after the FDA approved two drugs, Miplyffa and Aqneursa, to treat NPC.

“This is a life-threatening, very serious disorder, and the fact that we have more in our arsenal to really treat it has been just wonderful,” said Dr. Natasha Shur, a medical geneticist who works at Children’s National Hospital in Washington D.C.

Dr. Shur is part of Abby’s care team and believes this approval will improve the lives of patients across the country.

“Not only in their day but in their entire futures going forward and really helps maximize their potential and ability to interact,” Dr. Shur told 12 On Your Side.

The approval is paving a new path in Abby’s treatment plan.

“She actually has started Aqneursa, so we’re going to sort of start her on these two drugs,” said Garland Alvey. “All the while staying in her current trial, which we heard good news that there should be some movement there as well.”

The Alvey family will continue this journey until they find a cure, staying #AbbyStrong every step of the way.

“Where we thought Abby would just be sort of a footnote in NPC, this generation actually has a chance,” said Garland Alvey. “We’re never going to stop fighting.”

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