Cystic Fibrosis Weekly Update

Contents:

MRT5005 Gets FDA Rare Pediatric Disease Status as Adult Trial Faces Delay Due to COVID-19

Apr 01, 2020 07:00 am | David Melamed, PhD

Translate Bio‘s therapy MRT5005 has been given a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic fibrosis in children, while its ongoing clinical trial investigating the therapy in adult patients faces delays due to the COVID-19 pandemic, the company has announced. The designation is given to […]
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I’m Holding a Funeral for My Former Self

Mar 31, 2020 03:00 pm | Bailey Vincent

I am at a loss with loss. Yesterday, I lost my last remaining telecommuting job, my dance company (in the sense that we are now on a long-term hiatus), our show that was only weeks away (and has now been postponed), my keys, and my mind. If you’re like me, you’re sick of seeing sickness […]
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Vertex Assures Supply of CF Therapies, But COVID-19 Likely to Affect Trials

Mar 31, 2020 07:00 am | Mary Chapman

Vertex Pharmaceuticals is “highly confident” that it can continue to supply its approved cystic fibrosis (CF) therapies to patients worldwide, but expects at least some of its ongoing and planned clinical trials to be affected by the COVID-19 pandemic. Among likely changes to ongoing trials well into the dosing of patients — including a Phase 3 study of the recently approved […]
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Trikafta Therapy Prompted the Most Unexpected Emotions

Mar 30, 2020 03:00 pm | Wendy Caroline

The excitement and flurry around the new combination therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) brought a lot of hope to the CF community. This is a good thing. However, many complex emotions are not being addressed. Everyone expects a flood of challenging emotions when one transitions from healthy to dying, but what about the other way around? How […]
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