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Call the Midwife Evokes Cystic Fibrosis in a Simpler Time
Children with cystic fibrosis too young to pronounce the disease’s name call it 65 Roses. Previous posts bashing SyFy’s Helix and Dan Brown’s Inferno might suggest that I’m hypercritical of TV and...
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A Checklist for Gene Therapy From the UK Cystic Fibrosis Trial
A clinical trial is a series of hurdles, but not in parallel. Washington, D.C. I’m at the American Society of Gene and Cell Therapy annual meeting, one of my favorite conferences. The very...
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When Mutation Counters Infection: From Sickle Cell to Ebola
While pharmaceutical companies focus on drug discovery for Ebola virus disease, a powerful clue is coming from a rare “Jewish genetic disease” that destroys the brain. People with Niemann-Pick C1...
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New Miracle Drugs: What Would You Pay?
Levi Collazo, before and after taking Kalydeco for CF. Levi Collazo, a biology major at Southwestern Oklahoma State University, has cystic fibrosis (CF). “In the first photo, I was making fun of my own...
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When a Bad Animal Model is Good: Cystic Fibrosis
A “good” animal model is one that has the same symptoms of a disease that we do, right? Not always. Sometimes we can actually learn more when an animal is not a perfect model; their good health can...
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CF Rx: Exercise
We can’t change our genes – yet – but we can alter our environments to ease life with an inherited illness. Meet two young people whose love of dance, running, yoga, and more is helping them live with...
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Medical Success Stories: From Cystic Fibrosis to Diabetes
A skinny little boy, with mocha skin and curly black hair, lived in the apartment building next door when I was growing up in Brooklyn in the 1960s. I don’t remember his name, but I recall that he...
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Cystic Fibrosis Among Asians: Why Ethnicity-Based Genetic Testing is Obsolete
A hypothetical heterosexual couple living in the US or UK takes tests to learn if they are carriers of the more prevalent recessive diseases. They’re relieved to find out that cystic fibrosis (CF)...
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What Should Gene Therapy Cost?
By the time that the FDA’s advisory committee gave a near-instantaneous and unanimous thumbs-up for gene therapy to treat a form of hereditary blindness on October 12, tears were freely flowing....
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