This program will include a discussion of investigational agents not approved by the FDA for use in the US and data that were presented in abstract form These data should be considered preliminary until published in a peerreviewed journal ID: 674315
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Slide1
Advances in Cystic Fibrosis 2017Slide2
This program will include a discussion of investigational agents not approved by the FDA for use in the US, and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.Slide3
Cystic FibrosisSlide4
Treatment Landscape of Cystic FibrosisSlide5
Multidisciplinary Team ApproachSlide6
Preview of What's to ComeSlide7
Advances in Modulator
Therapies Targeting Common
and Uncommon CFTR MutationsSlide8
Previous CFTR Modulators
PotentiatorsSlide9
Previous CFTR Modulators
CorrectorsSlide10
TEZ/IVA for F508del
Homozygous PatientsSlide11
TEZ/IVA for F508del
Heterozygous
and Residual Function MutationSlide12
TEZ/IVA Efficacious and Well Tolerated
in
F508del Heterozygous Population Slide13
Closing CommentsSlide14
Novel Approaches Targeting
CFTR MutationsSlide15
CFTR CorrectorsSlide16
GLPG2222 Well ToleratedSlide17
The Potentiator GLPG2451 With and Without the CFTR Corrector GLP2222 in Healthy VolunteersSlide18
A Novel Type of Modulator Called
an AmplifierSlide19
The Novel CFTR Amplifier PTI-428
Shows PromiseSlide20
Nebulized QR-010: A Different Approach to Restore Mutant CFTR Function Using OligonucleotidesSlide21
SummarySlide22
Nursing Perspective on Quality
of Life and Disease BurdenSlide23
QoL After 3 Months of Ivacaftor -- Children's Hospital, PhiladelphiaSlide24
ResultsSlide25
TakeawaysSlide26
Caregiver Burden Due to Pulmonary Exacerbations in CFSlide27
FindingsSlide28
Concluding RemarksSlide29
Longer Term and Real-Life Outcome DataSlide30
We Know the Effects of FEV1
-- but What
Is the Mechanism? Slide31
Effect of LUM/IVA on Total Bronchiectasis and Air Trapping CT Scores in Children:
F508del
-CFTR MutationSlide32
Safety and Efficacy of LUM/IVA in Patients
With CF Homozygous for
F508del-CFTRSlide33
Effectiveness of LUM/IVA in Patients With
F508del
Homozygous CF Following FDA ApprovalSlide34
Modeling Long-Term Health Outcomes in Patients With CF Homozygous
F508del
Treated With LUM/IVASlide35
Concluding RemarksSlide36
AbbreviationsSlide37
Abbreviations (cont)