Progress in therapies for cystic fibrosis.

No abstract is available for this paper.

• Publication year

  2016

• Venue

  The Lancet Respiratory Medicine

• Publication date

  2016-08-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1016/S2213-2600(16)00023-0PMID 27053340
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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• 126 A phase 1 clinical study of CTX-4430 in cystic fibrosis patients

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• 139 OligoG fluorescent conjugation for localisation in a mucoid pseudomonal biofilm

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  2015cited by this paper
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  2014cited by this paper
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• Amiloride-Sensitive Sodium Channels and Pulmonary Edema

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