FDA Approves Kalydeco for Cystic Fibrosis Patients Ages 2 and Up

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The U.S. Food and Drug Administration (FDA) continues to provide new indications for Vertex’s Kalydeco (ivacaftor) for Cystic Fibrosis (CF) patients. Earlier today, it was approved for use in more than 600 patients ages 2 and older who have 1 of 5 residual function mutations that results in a splicing Continue reading FDA Approves Kalydeco for Cystic Fibrosis Patients Ages 2 and Up

New cystic fibrosis drug (Vertex)

https://www.ft.com/content/aa944954-13f4-11e7-b0c1-37e417ee6c76

Combination treatment offers safer option for patients suffering from deadly illness

Vertex Pharmaceuticals said that a combination of two of its medicines had successfully treated cystic fibrosis in a closely watched clinical trial, paving the way for regulators to approve a Continue reading New cystic fibrosis drug (Vertex)

Phase 2a CF corrector study

Galapagos doses first patient with novel CF corrector GLPG2222

Galapagos NV (Euronext & NASDAQ: GLPG) announces dosing of the first patient with cystic fibrosis (CF) Class III (F508del and a gating mutation like G551D) with novel CF corrector GLPG2222 as an add-on to Kalydeco®[1] in a Phase 2a study. Galapagos further announced the opening of an Investigational New Drug (IND) file with the US Food & Drug Administration for GLPG2222, triggering a $10 million milestone payment.
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Concert Pharmaceuticals Initiates Phase 2 Clinical Trial

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Concert Pharmaceuticals Initiates Phase 2 Clinical Trial Evaluating CTP-656 for the Treatment of Cystic Fibrosis

Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the initiation of a U.S.-based Phase 2 clinical trial evaluating CTP-656 Continue reading Concert Pharmaceuticals Initiates Phase 2 Clinical Trial

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016