<?xml version="1.0" encoding="ISO-8859-1"?><article xmlns:mml="http://www.w3.org/1998/Math/MathML" xmlns:xlink="http://www.w3.org/1999/xlink" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance">
<front>
<journal-meta>
<journal-id>0028-3746</journal-id>
<journal-title><![CDATA[Neumología y cirugía de tórax]]></journal-title>
<abbrev-journal-title><![CDATA[Neumol. cir. torax]]></abbrev-journal-title>
<issn>0028-3746</issn>
<publisher>
<publisher-name><![CDATA[Sociedad Mexicana de Neumología y Cirugía de Tórax; Instituto Nacional de Enfermedades Respiratorias Ismael Cosío Villegas; Sociedad Cubana de Neumología; Sociedad Paraguaya de Neumología; Sociedad Boliviana de Neumología.]]></publisher-name>
</publisher>
</journal-meta>
<article-meta>
<article-id>S0028-37462024000100006</article-id>
<article-id pub-id-type="doi">10.35366/118570</article-id>
<title-group>
<article-title xml:lang="en"><![CDATA[Cystic fibrosis transmembrane conductance regulator modulators and novel therapeutics for cystic fibrosis treatment]]></article-title>
<article-title xml:lang="es"><![CDATA[Moduladores de la conductancia de transmembrana de fibrosis quística y nuevos tratamientos para fibrosis quística]]></article-title>
</title-group>
<contrib-group>
<contrib contrib-type="author">
<name>
<surname><![CDATA[Bustamante]]></surname>
<given-names><![CDATA[Adriana Ester]]></given-names>
</name>
<xref ref-type="aff" rid="Aff"/>
<xref ref-type="aff" rid="Aaf"/>
</contrib>
</contrib-group>
<aff id="Af1">
<institution><![CDATA[,Clínica de Fibrosis Quística  ]]></institution>
<addr-line><![CDATA[Monterrey Nuevo León]]></addr-line>
<country>México</country>
</aff>
<pub-date pub-type="pub">
<day>00</day>
<month>03</month>
<year>2024</year>
</pub-date>
<pub-date pub-type="epub">
<day>00</day>
<month>03</month>
<year>2024</year>
</pub-date>
<volume>83</volume>
<numero>1</numero>
<fpage>6</fpage>
<lpage>12</lpage>
<copyright-statement/>
<copyright-year/>
<self-uri xlink:href="http://www.scielo.org.mx/scielo.php?script=sci_arttext&amp;pid=S0028-37462024000100006&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://www.scielo.org.mx/scielo.php?script=sci_abstract&amp;pid=S0028-37462024000100006&amp;lng=en&amp;nrm=iso"></self-uri><self-uri xlink:href="http://www.scielo.org.mx/scielo.php?script=sci_pdf&amp;pid=S0028-37462024000100006&amp;lng=en&amp;nrm=iso"></self-uri><abstract abstract-type="short" xml:lang="en"><p><![CDATA[Abstract: Cystic fibrosis (CF) is a hereditary, autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. To date, more than 2,000 mutations or variants in this gene have been described. Historically, the treatment of CF focused on clinically managing the manifestations and complications resulting from CFTR protein dysfunction. The discovery of the gene and the mutations causing this disease has led to the development of drugs known as CFTR modulators, which restore and optimize the function of the defective protein. The objective of this publication is to conduct a review of these new medications and their impact on lung function, nutritional status, quality of life, and patient survival, serving as an example of personalized medicine.]]></p></abstract>
<abstract abstract-type="short" xml:lang="es"><p><![CDATA[Resumen: La fibrosis quística es una enfermedad hereditaria, autosómica recesiva, causada por mutaciones en el gen de la proteína reguladora de conductancia de transmembrana de fibrosis quística (CFTR). A la fecha se han descrito más de 2,000 mutaciones o variantes en dicho gen. Históricamente el tratamiento de fibrosis quística estaba enfocado en el manejo clínico de las manifestaciones y complicaciones ocasionadas por la disfunción de dicha proteína. El descubrimiento del gen y de las mutaciones causantes de esta enfermedad ha permitido el desarrollo de fármacos conocidos como moduladores del CFTR que restauran y optimizan la función de la proteína defectuosa. El objetivo de esta publicación es llevar a cabo una revisión de estos nuevos medicamentos y su impacto sobre la función pulmonar, el estado nutricional, la calidad de vida y la supervivencia de los pacientes, constituyendo un ejemplo de medicina personalizada.]]></p></abstract>
<kwd-group>
<kwd lng="en"><![CDATA[cystic fibrosis]]></kwd>
<kwd lng="en"><![CDATA[cystic fibrosis transmembrane conductance regulator modulators]]></kwd>
<kwd lng="en"><![CDATA[personalized medicine]]></kwd>
<kwd lng="es"><![CDATA[fibrosis quística]]></kwd>
<kwd lng="es"><![CDATA[moduladores de conductancia de transmembrana de fibrosis quística]]></kwd>
<kwd lng="es"><![CDATA[medicina personalizada]]></kwd>
</kwd-group>
</article-meta>
</front><back>
<ref-list>
<ref id="B1">
<label>1</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Boucher]]></surname>
<given-names><![CDATA[RC]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Airway surface dehydration in cystic fibrosis: pathogenesis and therapy]]></article-title>
<source><![CDATA[Annu Rev Med]]></source>
<year>2007</year>
<volume>58</volume>
<page-range>157-70</page-range></nlm-citation>
</ref>
<ref id="B2">
<label>2</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Rowe]]></surname>
<given-names><![CDATA[SM]]></given-names>
</name>
<name>
<surname><![CDATA[Miller]]></surname>
<given-names><![CDATA[S]]></given-names>
</name>
<name>
<surname><![CDATA[Sorscher]]></surname>
<given-names><![CDATA[EJ]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Cystic fibrosis]]></article-title>
<source><![CDATA[N Engl J Med]]></source>
<year>2005</year>
<volume>352</volume>
<numero>19</numero>
<issue>19</issue>
<page-range>1992-2001</page-range></nlm-citation>
</ref>
<ref id="B3">
<label>3</label><nlm-citation citation-type="">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Project]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
</person-group>
<source><![CDATA[Clinical and Functional Translation of CFTR]]></source>
<year>2024</year>
</nlm-citation>
</ref>
<ref id="B4">
<label>4</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Elborn]]></surname>
<given-names><![CDATA[JS]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Cystic fibrosis]]></article-title>
<source><![CDATA[Lancet]]></source>
<year>2016</year>
<volume>388</volume>
<numero>10059</numero>
<issue>10059</issue>
<page-range>2519-31</page-range></nlm-citation>
</ref>
<ref id="B5">
<label>5</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Veit]]></surname>
<given-names><![CDATA[G]]></given-names>
</name>
<name>
<surname><![CDATA[Avramescu]]></surname>
<given-names><![CDATA[RG]]></given-names>
</name>
<name>
<surname><![CDATA[Chiang]]></surname>
<given-names><![CDATA[AN]]></given-names>
</name>
<name>
<surname><![CDATA[Houck]]></surname>
<given-names><![CDATA[SA]]></given-names>
</name>
<name>
<surname><![CDATA[Cai]]></surname>
<given-names><![CDATA[Z]]></given-names>
</name>
<name>
<surname><![CDATA[Peters]]></surname>
<given-names><![CDATA[KW]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations]]></article-title>
<source><![CDATA[Mol Biol Cell]]></source>
<year>2016</year>
<volume>27</volume>
<numero>3</numero>
<issue>3</issue>
<page-range>424-33</page-range></nlm-citation>
</ref>
<ref id="B6">
<label>6</label><nlm-citation citation-type="">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Foundation]]></surname>
<given-names><![CDATA[CF]]></given-names>
</name>
</person-group>
<source><![CDATA[2022 Patient Registry Annual Data Report]]></source>
<year>2022</year>
</nlm-citation>
</ref>
<ref id="B7">
<label>7</label><nlm-citation citation-type="">
<collab>European Cystic Fibrosis Society</collab>
<source><![CDATA[ECFS Patient Registry]]></source>
<year>2020</year>
</nlm-citation>
</ref>
<ref id="B8">
<label>8</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Scotet]]></surname>
<given-names><![CDATA[V]]></given-names>
</name>
<name>
<surname><![CDATA[L&#8217;Hostis]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
<name>
<surname><![CDATA[Férec]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[The changing epidemiology of cystic fibrosis: incidence, survival and impact of the CFTR Gene Discovery]]></article-title>
<source><![CDATA[Genes (Basel)]]></source>
<year>2020</year>
<volume>11</volume>
<numero>6</numero>
<issue>6</issue>
<page-range>589</page-range></nlm-citation>
</ref>
<ref id="B9">
<label>9</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Lopes-Pacheco]]></surname>
<given-names><![CDATA[M]]></given-names>
</name>
<name>
<surname><![CDATA[Sabirzhanova]]></surname>
<given-names><![CDATA[I]]></given-names>
</name>
<name>
<surname><![CDATA[Rapino]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
<name>
<surname><![CDATA[Morales]]></surname>
<given-names><![CDATA[MM]]></given-names>
</name>
<name>
<surname><![CDATA[Guggino]]></surname>
<given-names><![CDATA[WB]]></given-names>
</name>
<name>
<surname><![CDATA[Cebotaru]]></surname>
<given-names><![CDATA[L]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Correctors rescue CFTR mutations in nucleotide-binding domain 1 (NBD1) by modulating proteostasis]]></article-title>
<source><![CDATA[Chembiochem]]></source>
<year>2016</year>
<volume>17</volume>
<numero>6</numero>
<issue>6</issue>
<page-range>493-505</page-range></nlm-citation>
</ref>
<ref id="B10">
<label>10</label><nlm-citation citation-type="">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Inc]]></surname>
<given-names><![CDATA[VP]]></given-names>
</name>
</person-group>
<source><![CDATA[Kalydeco Prescribing information]]></source>
<year></year>
</nlm-citation>
</ref>
<ref id="B11">
<label>11</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Yu]]></surname>
<given-names><![CDATA[H]]></given-names>
</name>
<name>
<surname><![CDATA[Burton]]></surname>
<given-names><![CDATA[B]]></given-names>
</name>
<name>
<surname><![CDATA[Huang]]></surname>
<given-names><![CDATA[CJ]]></given-names>
</name>
<name>
<surname><![CDATA[Worley]]></surname>
<given-names><![CDATA[J]]></given-names>
</name>
<name>
<surname><![CDATA[Cao]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
<name>
<surname><![CDATA[Johnson]]></surname>
<given-names><![CDATA[JP]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Ivacaftor potentiation of multiple CFTR channels with gating mutations]]></article-title>
<source><![CDATA[J Cyst Fibros]]></source>
<year>2012</year>
<volume>11</volume>
<numero>3</numero>
<issue>3</issue>
<page-range>237-45</page-range></nlm-citation>
</ref>
<ref id="B12">
<label>12</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Robertson]]></surname>
<given-names><![CDATA[SM]]></given-names>
</name>
<name>
<surname><![CDATA[Luo]]></surname>
<given-names><![CDATA[X]]></given-names>
</name>
<name>
<surname><![CDATA[Dubey]]></surname>
<given-names><![CDATA[N]]></given-names>
</name>
<name>
<surname><![CDATA[Li]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
<name>
<surname><![CDATA[Chavan]]></surname>
<given-names><![CDATA[AB]]></given-names>
</name>
<name>
<surname><![CDATA[Gilmartin]]></surname>
<given-names><![CDATA[GS]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Clinical drug-drug interaction assessment of ivacaftor as a potential inhibitor of cytochrome P450 and P-glycoprotein]]></article-title>
<source><![CDATA[J Clin Pharmacol]]></source>
<year>2015</year>
<volume>55</volume>
<numero>1</numero>
<issue>1</issue>
<page-range>56-62</page-range></nlm-citation>
</ref>
<ref id="B13">
<label>13</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[McColley]]></surname>
<given-names><![CDATA[SA]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[A safety evaluation of ivacaftor for the treatment of cystic fibrosis]]></article-title>
<source><![CDATA[Expert Opin Drug Saf]]></source>
<year>2016</year>
<volume>15</volume>
<numero>5</numero>
<issue>5</issue>
<page-range>709-15</page-range></nlm-citation>
</ref>
<ref id="B14">
<label>14</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Ramsey]]></surname>
<given-names><![CDATA[BW]]></given-names>
</name>
<name>
<surname><![CDATA[Davies]]></surname>
<given-names><![CDATA[J]]></given-names>
</name>
<name>
<surname><![CDATA[McElvaney]]></surname>
<given-names><![CDATA[NG]]></given-names>
</name>
<name>
<surname><![CDATA[Tullis]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
<name>
<surname><![CDATA[Bell]]></surname>
<given-names><![CDATA[SC]]></given-names>
</name>
<name>
<surname><![CDATA[Drevínek]]></surname>
<given-names><![CDATA[P]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[A CFTR potentiator in patients with cystic fibrosis and the G551D mutation]]></article-title>
<collab>VX08-770-102 Study Group</collab>
<source><![CDATA[N Engl J Med]]></source>
<year>2011</year>
<volume>365</volume>
<numero>18</numero>
<issue>18</issue>
<page-range>1663-72</page-range></nlm-citation>
</ref>
<ref id="B15">
<label>15</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Taylor-Cousar]]></surname>
<given-names><![CDATA[JL]]></given-names>
</name>
<name>
<surname><![CDATA[Jain]]></surname>
<given-names><![CDATA[R]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Maternal and fetal outcomes following elexacaftor-tezacaftor-ivacaftor use during pregnancy and lactation]]></article-title>
<source><![CDATA[J Cyst Fibros]]></source>
<year>2021</year>
<volume>20</volume>
<numero>3</numero>
<issue>3</issue>
<page-range>402-6</page-range></nlm-citation>
</ref>
<ref id="B16">
<label>16</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Jain]]></surname>
<given-names><![CDATA[R]]></given-names>
</name>
<name>
<surname><![CDATA[Magaret]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Vu]]></surname>
<given-names><![CDATA[PT]]></given-names>
</name>
<name>
<surname><![CDATA[VanDalfsen]]></surname>
<given-names><![CDATA[JM]]></given-names>
</name>
<name>
<surname><![CDATA[Keller]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Wilson]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Prospectively evaluating maternal and fetal outcomes in the era of CFTR modulators: the MAYFLOWERS observational clinical trial study design]]></article-title>
<source><![CDATA[BMJ Open Respir Res]]></source>
<year>2022</year>
<volume>9</volume>
<numero>1</numero>
<issue>1</issue>
</nlm-citation>
</ref>
<ref id="B17">
<label>17</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Davies]]></surname>
<given-names><![CDATA[JC]]></given-names>
</name>
<name>
<surname><![CDATA[Wainwright]]></surname>
<given-names><![CDATA[CE]]></given-names>
</name>
<name>
<surname><![CDATA[Canny]]></surname>
<given-names><![CDATA[GJ]]></given-names>
</name>
<name>
<surname><![CDATA[Chilvers]]></surname>
<given-names><![CDATA[MA]]></given-names>
</name>
<name>
<surname><![CDATA[Howenstine]]></surname>
<given-names><![CDATA[MS]]></given-names>
</name>
<name>
<surname><![CDATA[Munck]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[; VX08-770-103 (ENVISION) Study Group. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation]]></article-title>
<source><![CDATA[Am J Respir Crit Care Med]]></source>
<year>2013</year>
<volume>187</volume>
<numero>11</numero>
<issue>11</issue>
<page-range>1219-25</page-range></nlm-citation>
</ref>
<ref id="B18">
<label>18</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Boyle]]></surname>
<given-names><![CDATA[MP]]></given-names>
</name>
<name>
<surname><![CDATA[De Boeck]]></surname>
<given-names><![CDATA[K]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect]]></article-title>
<source><![CDATA[Lancet Respir Med]]></source>
<year>2013</year>
<volume>1</volume>
<numero>2</numero>
<issue>2</issue>
<page-range>158-63</page-range></nlm-citation>
</ref>
<ref id="B19">
<label>19</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Flume]]></surname>
<given-names><![CDATA[PA]]></given-names>
</name>
<name>
<surname><![CDATA[Liou]]></surname>
<given-names><![CDATA[TG]]></given-names>
</name>
<name>
<surname><![CDATA[Borowitz]]></surname>
<given-names><![CDATA[DS]]></given-names>
</name>
<name>
<surname><![CDATA[Li]]></surname>
<given-names><![CDATA[H]]></given-names>
</name>
<name>
<surname><![CDATA[Yen]]></surname>
<given-names><![CDATA[K]]></given-names>
</name>
<name>
<surname><![CDATA[Ordoñez]]></surname>
<given-names><![CDATA[CL]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[; VX 08-770-104 Study Group. Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation]]></article-title>
<source><![CDATA[Chest]]></source>
<year>2012</year>
<volume>142</volume>
<numero>3</numero>
<issue>3</issue>
<page-range>718-24</page-range></nlm-citation>
</ref>
<ref id="B20">
<label>20</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Welsh]]></surname>
<given-names><![CDATA[MJ]]></given-names>
</name>
<name>
<surname><![CDATA[Smith]]></surname>
<given-names><![CDATA[AE]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Molecular mechanisms of CFTR chloride channel dysfunction in cystic fibrosis]]></article-title>
<source><![CDATA[Cell]]></source>
<year>1993</year>
<volume>73</volume>
<numero>7</numero>
<issue>7</issue>
<page-range>1251-4</page-range></nlm-citation>
</ref>
<ref id="B21">
<label>21</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Loo]]></surname>
<given-names><![CDATA[TW]]></given-names>
</name>
<name>
<surname><![CDATA[Bartlett]]></surname>
<given-names><![CDATA[MC]]></given-names>
</name>
<name>
<surname><![CDATA[Clarke]]></surname>
<given-names><![CDATA[DM]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Corrector VX-809 stabilizes the first transmembrane domain of CFTR]]></article-title>
<source><![CDATA[Biochem Pharmacol]]></source>
<year>2013</year>
<volume>86</volume>
<numero>5</numero>
<issue>5</issue>
<page-range>612-9</page-range></nlm-citation>
</ref>
<ref id="B22">
<label>22</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Fiedorczuk]]></surname>
<given-names><![CDATA[K]]></given-names>
</name>
<name>
<surname><![CDATA[Chen]]></surname>
<given-names><![CDATA[J]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Mechanism of CFTR correction by type I folding correctors]]></article-title>
<source><![CDATA[Cell]]></source>
<year>2022</year>
<volume>185</volume>
<numero>1</numero>
<issue>1</issue>
<page-range>158-168.e11</page-range></nlm-citation>
</ref>
<ref id="B23">
<label>23</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Taylor-Cousar]]></surname>
<given-names><![CDATA[JL]]></given-names>
</name>
<name>
<surname><![CDATA[Munck]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[McKone]]></surname>
<given-names><![CDATA[EF]]></given-names>
</name>
<name>
<surname><![CDATA[van der Ent]]></surname>
<given-names><![CDATA[CK]]></given-names>
</name>
<name>
<surname><![CDATA[Moeller]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Simard]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Tezacaftor-Ivacaftor in patients with cystic fibrosis homozygous for Phe508del]]></article-title>
<source><![CDATA[N Engl J Med]]></source>
<year>2017</year>
<volume>377</volume>
<numero>21</numero>
<issue>21</issue>
<page-range>2013-23</page-range></nlm-citation>
</ref>
<ref id="B24">
<label>24</label><nlm-citation citation-type="">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Inc]]></surname>
<given-names><![CDATA[VP]]></given-names>
</name>
</person-group>
<source><![CDATA[Prescribing information Trikafta]]></source>
<year></year>
</nlm-citation>
</ref>
<ref id="B25">
<label>25</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Fuchs]]></surname>
<given-names><![CDATA[HJ]]></given-names>
</name>
<name>
<surname><![CDATA[Borowitz]]></surname>
<given-names><![CDATA[DS]]></given-names>
</name>
<name>
<surname><![CDATA[Christiansen]]></surname>
<given-names><![CDATA[DH]]></given-names>
</name>
<name>
<surname><![CDATA[Morris]]></surname>
<given-names><![CDATA[EM]]></given-names>
</name>
<name>
<surname><![CDATA[Nash]]></surname>
<given-names><![CDATA[ML]]></given-names>
</name>
<name>
<surname><![CDATA[Ramsey]]></surname>
<given-names><![CDATA[BW]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group]]></article-title>
<source><![CDATA[N Engl J Med]]></source>
<year>1994</year>
<volume>331</volume>
<numero>10</numero>
<issue>10</issue>
<page-range>637-42</page-range></nlm-citation>
</ref>
<ref id="B26">
<label>26</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Pai]]></surname>
<given-names><![CDATA[VB]]></given-names>
</name>
<name>
<surname><![CDATA[Nahata]]></surname>
<given-names><![CDATA[MC]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Efficacy and safety of aerosolized tobramycin in cystic fibrosis]]></article-title>
<source><![CDATA[Pediatr Pulmonol]]></source>
<year>2001</year>
<volume>32</volume>
<numero>4</numero>
<issue>4</issue>
<page-range>314-27</page-range></nlm-citation>
</ref>
<ref id="B27">
<label>27</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[De Boeck]]></surname>
<given-names><![CDATA[K]]></given-names>
</name>
<name>
<surname><![CDATA[Munck]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Walker]]></surname>
<given-names><![CDATA[S]]></given-names>
</name>
<name>
<surname><![CDATA[Faro]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Hiatt]]></surname>
<given-names><![CDATA[P]]></given-names>
</name>
<name>
<surname><![CDATA[Gilmartin]]></surname>
<given-names><![CDATA[G]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation]]></article-title>
<source><![CDATA[J Cyst Fibros]]></source>
<year>2014</year>
<volume>13</volume>
<numero>6</numero>
<issue>6</issue>
<page-range>674-80</page-range></nlm-citation>
</ref>
<ref id="B28">
<label>28</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Volkova]]></surname>
<given-names><![CDATA[N]]></given-names>
</name>
<name>
<surname><![CDATA[Moy]]></surname>
<given-names><![CDATA[K]]></given-names>
</name>
<name>
<surname><![CDATA[Evans]]></surname>
<given-names><![CDATA[J]]></given-names>
</name>
<name>
<surname><![CDATA[Campbell]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
<name>
<surname><![CDATA[Tian]]></surname>
<given-names><![CDATA[S]]></given-names>
</name>
<name>
<surname><![CDATA[Simard]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries]]></article-title>
<source><![CDATA[J Cyst Fibros]]></source>
<year>2020</year>
<volume>19</volume>
<numero>1</numero>
<issue>1</issue>
<page-range>68-79</page-range></nlm-citation>
</ref>
<ref id="B29">
<label>29</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Barry]]></surname>
<given-names><![CDATA[PJ]]></given-names>
</name>
<name>
<surname><![CDATA[Plant]]></surname>
<given-names><![CDATA[BJ]]></given-names>
</name>
<name>
<surname><![CDATA[Nair]]></surname>
<given-names><![CDATA[A]]></given-names>
</name>
<name>
<surname><![CDATA[Bicknell]]></surname>
<given-names><![CDATA[S]]></given-names>
</name>
<name>
<surname><![CDATA[Simmonds]]></surname>
<given-names><![CDATA[NJ]]></given-names>
</name>
<name>
<surname><![CDATA[Bell]]></surname>
<given-names><![CDATA[NJ]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Effects of ivacaftor in patients with cystic fibrosis who carry the G551D mutation and have severe lung disease]]></article-title>
<source><![CDATA[Chest]]></source>
<year>2014</year>
<volume>146</volume>
<numero>1</numero>
<issue>1</issue>
<page-range>152-8</page-range></nlm-citation>
</ref>
<ref id="B30">
<label>30</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Hayes]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
<name>
<surname><![CDATA[McCoy]]></surname>
<given-names><![CDATA[KS]]></given-names>
</name>
<name>
<surname><![CDATA[Sheikh]]></surname>
<given-names><![CDATA[SI]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Improvement of sinus disease in cystic fibrosis with ivacaftor therapy]]></article-title>
<source><![CDATA[Am J Respir Crit Care Med]]></source>
<year>2014</year>
<volume>190</volume>
<numero>4</numero>
<issue>4</issue>
<page-range>468</page-range></nlm-citation>
</ref>
<ref id="B31">
<label>31</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Hayes]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
<name>
<surname><![CDATA[Warren]]></surname>
<given-names><![CDATA[PS]]></given-names>
</name>
<name>
<surname><![CDATA[McCoy]]></surname>
<given-names><![CDATA[KS]]></given-names>
</name>
<name>
<surname><![CDATA[Sheikh]]></surname>
<given-names><![CDATA[SI]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Improvement of hepatic steatosis in cystic fibrosis with ivacaftor therapy]]></article-title>
<source><![CDATA[J Pediatr Gastroenterol Nutr]]></source>
<year>2015</year>
<volume>60</volume>
<numero>5</numero>
<issue>5</issue>
<page-range>578-9</page-range></nlm-citation>
</ref>
<ref id="B32">
<label>32</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Sergeev]]></surname>
<given-names><![CDATA[V]]></given-names>
</name>
<name>
<surname><![CDATA[Chou]]></surname>
<given-names><![CDATA[FY]]></given-names>
</name>
<name>
<surname><![CDATA[Lam]]></surname>
<given-names><![CDATA[GY]]></given-names>
</name>
<name>
<surname><![CDATA[Hamilton]]></surname>
<given-names><![CDATA[CM]]></given-names>
</name>
<name>
<surname><![CDATA[Wilcox]]></surname>
<given-names><![CDATA[PG]]></given-names>
</name>
<name>
<surname><![CDATA[Quon]]></surname>
<given-names><![CDATA[BS]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[The extrapulmonary effects of cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis]]></article-title>
<source><![CDATA[Ann Am Thorac Soc]]></source>
<year>2020</year>
<volume>17</volume>
<numero>2</numero>
<issue>2</issue>
<page-range>147-54</page-range></nlm-citation>
</ref>
<ref id="B33">
<label>33</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Rowe]]></surname>
<given-names><![CDATA[SM]]></given-names>
</name>
<name>
<surname><![CDATA[McColley]]></surname>
<given-names><![CDATA[SA]]></given-names>
</name>
<name>
<surname><![CDATA[Rietschel]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
<name>
<surname><![CDATA[Li]]></surname>
<given-names><![CDATA[X]]></given-names>
</name>
<name>
<surname><![CDATA[Bell]]></surname>
<given-names><![CDATA[SC]]></given-names>
</name>
<name>
<surname><![CDATA[Konstan]]></surname>
<given-names><![CDATA[MW]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[; VX09-809-102 Study Group. Lumacaftor/Ivacaftor treatment of patients with cystic fibrosis heterozygous for F508del-CFTR]]></article-title>
<source><![CDATA[Ann Am Thorac Soc]]></source>
<year>2017</year>
<volume>14</volume>
<numero>2</numero>
<issue>2</issue>
<page-range>213-9</page-range></nlm-citation>
</ref>
<ref id="B34">
<label>34</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Rowe]]></surname>
<given-names><![CDATA[SM]]></given-names>
</name>
<name>
<surname><![CDATA[Daines]]></surname>
<given-names><![CDATA[C]]></given-names>
</name>
<name>
<surname><![CDATA[Ringshausen]]></surname>
<given-names><![CDATA[FC]]></given-names>
</name>
<name>
<surname><![CDATA[Kerem]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
<name>
<surname><![CDATA[Wilson]]></surname>
<given-names><![CDATA[J]]></given-names>
</name>
<name>
<surname><![CDATA[Tullis]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Tezacaftor-Ivacaftor in residual-function heterozygotes with cystic fibrosis]]></article-title>
<source><![CDATA[N Engl J Med]]></source>
<year>2017</year>
<volume>377</volume>
<numero>21</numero>
<issue>21</issue>
<page-range>2024-35</page-range></nlm-citation>
</ref>
<ref id="B35">
<label>35</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Middleton]]></surname>
<given-names><![CDATA[PG]]></given-names>
</name>
<name>
<surname><![CDATA[Mall]]></surname>
<given-names><![CDATA[MA]]></given-names>
</name>
<name>
<surname><![CDATA[Drevinek]]></surname>
<given-names><![CDATA[P]]></given-names>
</name>
<name>
<surname><![CDATA[Lands]]></surname>
<given-names><![CDATA[LC]]></given-names>
</name>
<name>
<surname><![CDATA[McKone]]></surname>
<given-names><![CDATA[EF]]></given-names>
</name>
<name>
<surname><![CDATA[Polineni]]></surname>
<given-names><![CDATA[D]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[; VX17-445-102 Study Group. Elexacaftor-Tezacaftor-Ivacaftor for cystic fibrosis with a single Phe508del Allele]]></article-title>
<source><![CDATA[N Engl J Med]]></source>
<year>2019</year>
<volume>381</volume>
<numero>19</numero>
<issue>19</issue>
<page-range>1809-19</page-range></nlm-citation>
</ref>
<ref id="B36">
<label>36</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Heijerman]]></surname>
<given-names><![CDATA[HGM]]></given-names>
</name>
<name>
<surname><![CDATA[McKone]]></surname>
<given-names><![CDATA[EF]]></given-names>
</name>
<name>
<surname><![CDATA[Downey]]></surname>
<given-names><![CDATA[DG]]></given-names>
</name>
<name>
<surname><![CDATA[Van Braeckel]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
<name>
<surname><![CDATA[Rowe]]></surname>
<given-names><![CDATA[SM]]></given-names>
</name>
<name>
<surname><![CDATA[Tullis]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[; VX17-445-103 Trial Group. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial]]></article-title>
<source><![CDATA[Lancet]]></source>
<year>2019</year>
<volume>394</volume>
<numero>10212</numero>
<issue>10212</issue>
<page-range>1940-8</page-range></nlm-citation>
</ref>
<ref id="B37">
<label>37</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[He]]></surname>
<given-names><![CDATA[R]]></given-names>
</name>
<name>
<surname><![CDATA[Lin]]></surname>
<given-names><![CDATA[F]]></given-names>
</name>
<name>
<surname><![CDATA[Deng]]></surname>
<given-names><![CDATA[Z]]></given-names>
</name>
<name>
<surname><![CDATA[Yu]]></surname>
<given-names><![CDATA[B]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with Phe508del mutation: Evidence from randomized controlled trials]]></article-title>
<source><![CDATA[SAGE Open Med]]></source>
<year>2024</year>
<volume>12</volume>
<page-range>20503121231225874</page-range></nlm-citation>
</ref>
<ref id="B38">
<label>38</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Saluzzo]]></surname>
<given-names><![CDATA[F]]></given-names>
</name>
<name>
<surname><![CDATA[Riberi]]></surname>
<given-names><![CDATA[L]]></given-names>
</name>
<name>
<surname><![CDATA[Messore]]></surname>
<given-names><![CDATA[B]]></given-names>
</name>
<name>
<surname><![CDATA[Loré]]></surname>
<given-names><![CDATA[NI]]></given-names>
</name>
<name>
<surname><![CDATA[Esposito]]></surname>
<given-names><![CDATA[I]]></given-names>
</name>
<name>
<surname><![CDATA[Bignamini]]></surname>
<given-names><![CDATA[E]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[CFTR modulator therapies: potential impact on airway infections in cystic fibrosis]]></article-title>
<source><![CDATA[Cells]]></source>
<year>2022</year>
<volume>11</volume>
<numero>7</numero>
<issue>7</issue>
<page-range>1243</page-range></nlm-citation>
</ref>
<ref id="B39">
<label>39</label><nlm-citation citation-type="journal">
<person-group person-group-type="author">
<name>
<surname><![CDATA[Zampoli]]></surname>
<given-names><![CDATA[M]]></given-names>
</name>
<name>
<surname><![CDATA[Morrow]]></surname>
<given-names><![CDATA[BM]]></given-names>
</name>
<name>
<surname><![CDATA[Paul]]></surname>
<given-names><![CDATA[G]]></given-names>
</name>
</person-group>
<article-title xml:lang=""><![CDATA[Real-world disparities and ethical considerations with access to CFTR modulator drugs: Mind the gap!]]></article-title>
<source><![CDATA[Front Pharmacol]]></source>
<year>2023</year>
<volume>14</volume>
<page-range>1163391</page-range></nlm-citation>
</ref>
</ref-list>
</back>
</article>
