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Patients with rare diseases are worried about a move to cut drug prices

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Patients with rare diseases are worried about a move to cut drug prices


Trikafta, as used by Sabrina Walker with cystic-fibrosis has changed her life.

Before starting the drug, she spent weeks in hospital until antibiotics cleared up the infection in her lungs. She wore a vest every day that would shake her body and loosen up the mucus.

In 2019, she had a particularly bad flare up, also known as a “pulmonary exacerbation”, which caused her to cough blood. She was then put on the new breakthrough drug.

She said that her lung function had increased by 20% in a month and her health was improving. Trakafta helped her avoid three or four hospitalizations per year. In the four years she’s been taking the medication, only one hospitalization has occurred.

“I was spending hours a day doing airway clearance and breathing treatments, and that has been significantly reduced,” said the 37-year-old Erie, Colorado, mother. “I’ve gained hours back in my day.”

Now, she works full-time and runs in Colorado’s thin air. Many other patients have experienced similar results with drug therapy. They were able to return to their regular lives, and some even removed themselves from the waiting list for a lung-transplant. Walker and scores of…



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