Cystic Fibrosis Weekly Update

Contents:

Planned CHOICES Trial in Rare CF Mutations Given Regulatory Support

Apr 15, 2020 07:00 am | Ines Martins, PhD

Proteostasis Therapeutics reported receiving scientific advice for its CHOICES clinical trial program, which is to test its personalized triple combination for cystic fibrosis (CF) patients without available treatment options, from the Dutch Medicines Evaluation Board. Regulatory officials outlined necessary steps in this planned, pivotal trial that would support approval of the company’s CFTR modulators — dirocaftor, posenacaftor, […]
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I Am Not a Pre-existing Condition

Apr 14, 2020 09:00 am | Bailey Vincent

pre-existing condition, perspective, slowing down

My life feels like a walking disclaimer lately. It’s as though everything I do or say or write should come with the headline proclaiming “I have a pre-existing condition.” These words are everywhere; I can’t seem to escape the phrase. It almost feels like a justification, doesn’t it? As if saying “but they had a […]
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Maybe I’m Not Fine. Are You?

Apr 14, 2020 09:00 am | Brad Dell

I’ve been bored during my six weeks of isolation. But sometimes boring is a synonym for peaceful. Yes, it’s been a peaceful time of silence and introspection. For example, in all my sitting around, I realized something really cool: I’m an amazing liar! I’m so good at it that sometimes I don’t even realize I’m […]
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Arrowhead Asks to Open Phase 1/2 Trial into Inhaled RNAi Therapy

Apr 14, 2020 07:00 am | Patricia Inacio, PhD

Arrowhead Pharmaceuticals has filed for approval to launch a Phase 1/2a clinical trial in New Zealand testing its candidate inhaled therapy for cystic fibrosis (CF), called ARO-ENaC, the company announced. ARO-ENaC is an investigational RNA therapy designed to lower the production of the epithelial sodium channel alpha subunit (αENaC) in the lungs of CF patients. ENaC transports sodium and is hyper-activated […]
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The US Healthcare System Desperately Needs Change

Apr 13, 2020 03:00 pm | Wendy Caroline

I am drained. My current situation astonishes me. The United States healthcare system must change. Over the past month, I’ve dealt with some severe and unusual health insurance issues. Before I continue, I want to make it clear that this is not an attack on our healthcare workers or any one person, nor am I […]
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