Cystic fibrosis - Ten promising therapeutic approaches in the current era of care.
Ranjani SomayajiDave P NicholsScott Cameron BellPublished in: Expert opinion on investigational drugs (2020)
Since the discovery of the CF gene, CFTR modulators have provided a hallmark of success, even though it was thought not previously possible. This has led to reinvigorated efforts and innovations in treatment approaches and care delivery. Numerous challenges remain because of genetic and phenotypic heterogeneity, access issues, and therapeutic costs, but the collaborative approach between stakeholders for continued innovation fuels optimism. Abbreviations: CF cystic fibrosis; CFF Cystic Fibrosis Foundation (USA); CFTR cystic fibrosis transmembrane regulator; CRISPR clustered regularly interspaced short palindromic repeats; COX cyclo oxygenase; FDA US Food and Drug Administration; FEV1% forced expiratory volume in one second % predicted; F508del deletion of phenylalanine (F) in the 508th position (most common mutation); G551D substitution of the amino acid glycine by aspartate at position 551 in the nucleotide binding domain-1 of the CFTR gene; LMIC low- and middle-income country; LTB4 leukotriene B4; MDT multi-disciplinary care team; NO nitric oxide; NSAIDs non-steroidal anti-inflammatory drugs; SLPI secretory leukocyte protease inhibitor.
Keyphrases
- cystic fibrosis
- anti inflammatory drugs
- quality improvement
- pseudomonas aeruginosa
- palliative care
- lung function
- genome wide
- healthcare
- nitric oxide
- copy number
- small molecule
- amino acid
- drug administration
- pain management
- affordable care act
- mental health
- single cell
- gene expression
- hydrogen peroxide
- intensive care unit
- nitric oxide synthase
- human health