From a name for an unnamed illness to a decade of modulators — the arc this lab supports, in four beats.
Research, not medical advice Sources and dates on each panel
In 1938, Dorothy Andersen published the first characterization of cystic fibrosis. Parents founded the Cystic Fibrosis Foundation in 1955. Foundation-supported scientists discovered the CFTR gene and its protein product in 1989.
Names: CFF history timeline at cff.org/about-us/our-history.
The Foundation describes 1955 as a time when people born with CF were not expected to reach elementary school. Its history page now describes people living into their 30s, 40s, and beyond. This is a historical account, not an individual prognosis. A 2022 estimate put the population at 40,000 in the U.S. and 105,000 worldwide.
Source: CFF history timeline, checked October 4, 2026. The population figures are dated estimates, not current counts.
1993 — Pulmozyme® became the first drug developed specifically for CF to receive FDA approval. 2019 — Trikafta®, a triple-combination CFTR modulator, received FDA approval. 2024 — Alyftrek®, an oral CFTR modulator, followed. These are U.S. approval milestones, not statements of individual eligibility or access; modulators do not address every CFTR variant.
This lab does not list dosing, eligibility, or side-effect claims — clinical decisions belong between a person and their care team. Alyftrek's oral formulation is documented in its U.S. prescribing information (label updated August 26, 2026; checked October 4, 2026).
The Foundation's mission — to find a cure, improve care, and support the CF community — frames the lab's own scope. What remains open: who is still underserved by existing modulators, what the next therapy class looks like, and how a person with CF lives day to day. This page is a research aid, not medical advice.
For the full history, including the 1955 founding story, read the CFF pages linked above. For community, the 65-rose field at the top of this page is our independent tribute.
See the protein
A 3D drawing of the CFTR protein, built from four published structures. It shows where amino acid 508 sits in two of the structures, and the gap where it is missing in the other two. It does not show where the pore or the gate is.
WebGL2 Cryo-EM maps:
Genetics primer
Access
Modulators do not reach everyone who has CF. The third panel above says it plainly: they do not address every CFTR variant. But a genotype is not the only thing that decides who can get a treatment. Whether a country has licensed a modulator, and whether its health system pays for it, decide too. Access is a matter of systems, prices and decisions, not of anyone's worth.
Europe. The European Cystic Fibrosis Society Patient Registry (ECFSPR) report for 2024 maps, for each modulator, whether it was licensed and reimbursed by the national health service in each of the countries taking part in the registry that year. The information was supplied by each country's nominated representative. Source: ECFSPR Annual Data Report 2024, chapter nine, from the registry's annual reports page.
Canada. The Canadian Cystic Fibrosis Registry's report for 2024 notes that people who became eligible after an expansion in July 2024 may not have had access straight away. The report gives two possible reasons: delays in drug coverage, and a person's own choice not to start treatment. Source: Canadian Cystic Fibrosis Registry Annual Data Report 2024, from the registry's page.
United States. Within the U.S. registry the picture is uneven too. The 2024 report gives data for adolescents and adults counted as not eligible for a modulator by age or genotype; that count includes people who are eligible only for the newly approved Alyftrek. Of that group, 14.9 percent are Black and 23.7 percent are Hispanic, against 3.4 percent and 10.3 percent of the whole registry. These are shares of one group, not rates of anything, and they say nothing about why. Source: Cystic Fibrosis Foundation Patient Registry 2024 Annual Data Report, from the Foundation's registry page.
The three reports were read for this page on 2026-10-05. Each registry counts its own population in its own way, and we compare no figure from one registry with another.
We do not have a verified count of how many people with CF worldwide cannot get a modulator, and we will not guess one. The only worldwide figure on this page is the dated 2022 population estimate in the second panel above. The lab does not speak for anyone and has no way to change any of this. What it can do is show the evidence plainly and point to the people who gather it.
Peer-reviewed papers on access and inequity, found with the lab's evidence tool. The list includes work on prices and production costs; we say no more about cost than that title does.
Limits
It gives no dosing, eligibility or side-effect information. It does not predict any person's outcome. The population figures above are dated estimates, and the approval years are U.S. milestones, not statements about who can get a treatment.
Where we do not know something yet, we say so. Each fact on this page carries its source and the date we checked it.
Where we stand
A tribute
The roses drifting behind this page, sixty-five of them, are an independent tribute to the Cystic Fibrosis Foundation's “65 Roses”. The name comes from a child's pronunciation of cystic fibrosis, not from a founding patient count. It is a Foundation trademark, not this lab's emblem, and the tribute is not an endorsement. The Foundation tells the story itself: the 65 Roses story.
Support
Giving is optional. It goes to the Foundation, not to this lab, and we are not affiliated with it.
Give to CF research through the Foundation (Washington chapter)
Tracking ~6–12% waiting across 5 registries and 57,288 ECFSPR patients.
4ee7fcc1c002159041ec3c480bed1c5666adb8cc52c8d5ca65a6eb128c243b40 · ecfspr_v0_1_audit.json ↗
Nonsense rescue (G542X, W1282X) via mRNA protection, tRNA readthrough, and folding.
Six research threads across nineteen verified milestones toward CFTR restoration.
Interactive constellation map loading.
Explore the standalone interactive tree with guided quests →
All CFTR genotypes across four research tracks.
LNP stealth (NCT05668741).
SMG1i & sup-tRNA (NM_000492.4).
ETD001 & TMEM16A (7.8 μm ASL).
6MSM, 5UAK, 8EIQ, 8EJ1.
OprM & LPS selection.
HIT-CF (NCT06468527).
982 patients (NCT02781610).
940 trials, 23 classes.
1,772 studies, 0 retractions.
