A team of Portland doctors delivered potentially lifesaving treatment to a girl battling a rare genetic disease.

Calypso, 5, is said to be the first in the state to receive a new FDA approved drug called Spinraza. Doctors are crediting her mother for fighting to put her first in line.

Calypso was diagnosed with Spinal Muscular Atrophy years ago. A diagnosis that means her brain stopped telling her muscles to move.

"Calypso was a week away from 9-months old before she was first hospitalized," said mom Celia Vander Velden. "I was just torn apart, I couldn't think, I think I asked to sit down, it was hard. Then I just looked over at her and I thought, 'OK well I can't do that in front of her.'"

Calypso weakens little by little every day, though doctors say she still understands what's going on around her.

"She is ventilator dependent, she relies on machines to breathe, to feed her, to suction her mouth out so she doesn't choke on her saliva," said Vander Velden.

The genetic disease is considered incurable, but there are ways to treat symptoms. Vander Velden became fixated on that treatment and a new drug called Spinraza.

"As far as Spinraza goes this has been a journey, we first heard about it when she was two," Vander Velden said.

Back then, Spinraza wasn't FDA approved. So, Vander Velden fought to get Calypso into clinical trials. A fight that took her from home in Douglas County to Randall Children's Hospital in Portland.

"I know many families can't wait," said RCH Pediatric Pulmonologist Mariam Ischander. "Sometimes there's no time, sometimes we lose a child to a cold."

Ischander became invested in Vander Velden's mission to get her daughter into a clinical trial. Until, unexpectedly, Spinraza was approved.

"Sure enough it was a Christmas gift. It was the best celebration ever on December 23," said Ischander.

Working with her colleagues at the hospital's new muscular dystrophy clinic, Ischander got her hands on the drug in March. Calypso was the first person in Oregon to receive treatment, according to staff at Randall Children's Hospital.

"I placed the needle into her spine to draw off spinal fluid and once we drew out the appropriate amount, we injected the medication through that same needle into the spine," said George Anadiotis Medical Director of Chemical and Biochemical Genetics.

Doctors say the drug works like a patch in computer code and is designed to stimulate proteins in the body that help muscles move and grow.

"This is a total game changer," said John McQueston Medical Directory for Respiratory Therapy at RCH. "This is going to prevent the kind of slow decline that we saw in the past."

New movement in Calypso's hand is a sign the drug may be working.

"I would say it's enough to have the hope, it was an honor after all those kids and how many kids we lost, to see this," said Ischander. "I honestly feel it's a great honor."

Hope Vander Velden holds on to.

"The first goal in my heart is to stop her smile from going away," said Vander Velden. "We're not biding our time, we're fighting and waiting for the next step. She's here and this is maintenance until the cure comes."

Doctors say they'll need six months before they'll be able to officially confirm if the drug is working with a neuro exam.

Anyone interested in helping the Vander Velden family can visit: www.youcaring.com/calypso-odessa-hope-vv-643592

Copyright 2017 KPTV-KPDX Broadcasting Corporation. All rights reserved.


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