7-Year Old Girl Has Almost Instant Transformation After Given ‘Miracle Drug’ For Cystic Fibrosis Treatment

Shields Gazette

A 7-year old girl named Harriet Corr that would often have painful and constant coughing fits has been given a second chance at life, all due to a medication that her mom is now calling ‘a miracle drug.’

Little Harriet has the debilitating lung disease called cystic fibrosis (CR), which is an incredibly tough and complicated disease that disrupts the body’s ability to produce particular fluids, which as a result, causes persistent bacterial infections within the lungs, as well as other metabolic issues like diabetes. Sadly, it can be both complex and demanding to treat and at times, it can be fatal as well.

Harriet’s mom, Emma Corr – from Tyne and Wear in the north of England – knew that her daughter’s health was in decline from the very beginning of her life, and despite the fact that that she was just seven years old, she took it upon herself to beg for her daughter to be granted access to a drug named Orkambi, which is available in the United States, for all those seven years that her beloved daughter has been alive.

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Emma teamed up with a bunch of parents whose children also have cystic fibrosis in order to get their government to get the drug available on the NHS. Not only was Orkambi made available to them by October 2019, another medication named Kaftrio, was also given the green light by February 2022.

Although the Orkambi medication didn’t work for Harriet’s condition, when she began taking Kaftrio in February 2022, it was like there was an “overnight” transformation in little Harriet’s health.

These days, the young schoolgirl can run around without falling into coughing fits or losing her breath, as well as keep up with other kids her own age. Moreover, she’s gained weight and now doesn’t stay up all night due to incessant coughing.

Amazingly, her lung capacity is at 120%, which is almost double of the scariest time in her life when it was just 67%.

According to her mom Emma, “It’s not an exaggeration to say I didn’t sleep for the first seven years of Harriet’s life. I would sit up all night by her side as she would cough.”

She added, “Campaigning to get the drugs available on the NHS is what pushed me through as I would email MPs throughout the night. I get scared speaking about it in case I jinx it, but it is absolutely amazing to see how far Harriet has come from this time last year.”

Most people get their cystic fibrosis diagnosis very early in life, but because it’s such a serious and oftentimes, fatal, diagnosis, most doctors consider it protocol to warn parents about being emotionally prepared to lose their children before they even reach 10 years old.

During the month of August in 2021, Harriet’s health was at an all-time low, making Emma fear the worst. But as soon as Harriet began taking Kaftrio, it was like her health basically transformed overnight.

The way Kaftrio works is by ‘acting as a stand in for the body’s CFTR genes that regulate mucus and digestive fluid density, viscosity, and production, that are mutated in the disease.’ While one of the main active ingredients happens to improve the activity of the defective genes, the second one manages to increase their number.

As shared by Emma, “She coughed for 10 minutes and then never coughed again. Harriet is now like a new little girl, looking back at photos the transformation is phenomenal.”

Another plus is the fact that Nancy, Harriet’s older sister, feels as if she finally has her baby sister back since Harriet would spend such long periods of time being hospitalized for her condition.

Before she began her treatment, even a slight common cold could result in Harriet being brought to the hospital but now her immune system is finally fighting off illnesses the way its supposed to.

Emma went on to share, “She can sleep through the night and her older sister is so happy to have her little sister back. She’s a really big fun character and loves to be the center of attention. She used to struggle with her coughing whilst dancing, swimming and playing football but now nothing is holding her back.”