24.04.2026
We received good news from the Public Union “Rare Diseases of Ukraine”: The Ministry of Health of Ukraine has updated the composition of the group of experts and specialists involved in the work of the Permanent Working Group of the Ministry of Health on sectoral support for procurement in specific areas. From now on, the group includes representatives of the Public Union “Rare Diseases of Ukraine”:
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04.04.2026
Dear colleagues, patients, parents, and caregivers of people with cystic fibrosis, I would like to share important news that continues our collective efforts to support those who are currently facing extremely difficult life circumstances. With the approval of GlobalGiving, the “Orphan Tits” Foundation (Orphanni Synytsi) will continue in 2026 its program supporting charitable projects aimed at helping orphan patients in Ukraine.
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26.03.2026
In the context of ongoing challenges faced by our country and the healthcare system of Ukraine, the Association continues to fulfill its mission: to provide support to patients and healthcare professionals, to contribute to improving the quality of medical care, and to represent the interests of the field in cooperation with government authorities, international organizations, and partner institutions.
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16.03.2026
Fascinating and educational facts from the world of cystic fibrosis:
- a book has been published that describes the journey from the description of the first cases of cystic fibrosis in 1930 to modulator therapy “Breath from Salt” 👉👉 over 100 years patients and parents have made a breakthrough
- in patients with CF who ended up in prison, body mass index and respiratory indicators improved. A daily schedule and discipline matter
10.03.2026
From March 5 to 7, 2026, leading specialists gathered in Tbilisi for the 13th South Eastern European Cystic Fibrosis Conference (SEEC 2026). Among the many participants were doctors from Ukraine. They listened, learned, shared their experience, and brought home valuable knowledge from the world of cystic fibrosis. Today, we are pleased to give our doctors the floor and invite you to read their impressions and conclusions from this important event.
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18.02.2026
Every year, on the last day of February, the world unites to draw attention to people living with rare diseases. On February 27, we invite you to a powerful conference dedicated to current issues of orphan medicine in Ukraine. The event will be held in a hybrid format — offline in Kyiv and online for everyone who cannot attend in person.
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31.12.2025
Dear parents, patients, doctors, and our entire community! 💙
We welcome this New Year in a reality where the war has been going on for its fourth year. A reality of loss, anxiety, darkness, and waiting. But also a reality of extraordinary strength, humanity, and unity.
For families living with cystic fibrosis, this war has doubled every challenge. Treatment, hospitals, travel, fear for children and loved ones, life between air-raid sirens… And at the same time—a daily fight for breath, for life, for the future.
19.11.2025
Italian genetic researchers have developed an experimental drug called 3b, which, when taken together with CFTR modulators, creates a powerful dual effect in the treatment of cystic fibrosis.In early laboratory trials, compound 3b not only helped restore the function of the CFTR protein, whose damage is a hallmark of cystic fibrosis, but also inhibited the growth of some of the most common respiratory viruses. It is known that viruses—especially rhinoviruses, which cause the common cold—lead to a deterioration of lung function in people with cystic fibrosis.
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13.10.2025
In France, researchers compared rates of serious liver complications before and after starting treatment with Caftrio (elexacaftor, tezacaftor, and ivacaftor), which is sold in the United States as Tricafta. The study found that the risk of serious liver problems among patients dropped by more than 70% in the years after the triple combination therapy was approved.
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06.10.2025
Dear doctors! We sincerely congratulate you on International Doctor's Day! Your daily work is not just a profession, but a calling that requires deep knowledge, patience, humanity and a big heart. Special thanks to those who have dedicated themselves to helping patients with cystic fibrosis - you are on the forefront of the fight for the quality and duration of life of every child and adult with this diagnosis.
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19.09.2025
A German study found that the drug Caftrio (a combination of elexacaftor, tezacaftor, and ivacaftor, sold under the trade name Tricaft in the U.S.) may help adults with cystic fibrosis engage in more high-intensity exercise, although regular exercise remains key for all patients.
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09.09.2025
September 8 is World Cystic Fibrosis Day, a special date when we remember the strength and courage of our community, rejoice in what we have already achieved, and hope for a better future. Patients, their families, doctors, scientists, and everyone who helps — together we carry the knowledge and faith that everything will be fine.
Cysic fibrosis (CF) is a genetic disease that affects the respiratory and digestive systems. Hundreds of people with CF live in Ukraine, and each of them is a true fighter. Every day they undergo procedures, take medications, and do not give up, despite all the challenges.
19.08.2025
Antibiotics are often ineffective in chronic lung infections in CF, especially when Pseudomonas aeruginosa colonizes the respiratory tract. This bacterium has become resistant to modern antibiotics over time. A new approach to treating CF is based on the use of phage therapy with a nebulized three-component cocktail, BX004-A, which acts against Pseudomonas aeruginosa by infecting and killing the bacterium like a virus.
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27.07.2025
BETHESDA, Md. (July 16, 2025) – The Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of gene-editing therapies. Prime Medicine uses prime editing technology, a highly precise DNA editing process. The new investment focuses on developing a prime-editing therapy for the G542X mutation, one of the cystic fibrosis mutations for which there is no cure.
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13.07.2025
Vertex Pharmaceuticals’ new targeted drug Alyftrek, a triplet that combines vanzacaftor, tezacaftor and deutivacaftor, will be approved by the European Commission (EC) following a positive recommendation in April 2025 by the European Medicines Agency (EMA).
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25.06.2025
Rapid reduction in infection visits and antibiotic use in people with cystic fibrosis after starting Elexacaftor-Tezacaftor-Ivacaftor.
In the article at the link, you can read about the study of the effectiveness of ELX/TEZ/IVA (or Tricaft) in cystic fibrosis.
This article presents the results of a real-world study of the effectiveness of the triple combination of CFTR modulators — elexacaftor-tezacaftor-ivacaftor (ELX/TEZ/IVA) — in patients with cystic fibrosis (CF).
03.06.2025
We are pleased to announce that today another Reference Orphan Center in Ukraine was officially approved - KNP "Zaporizhzhia Regional Clinical Hospital"! ⠀
The center received recognition in three areas:
1. Cystic fibrosis (CF).
2. Autoimmune diseases (JURA).
3. Pituitary dwarfism.
17.05.2025
Long-term efficacy of dual CFTR modulator treatment of cystic fibrosis LUM/IVA, Orkambi TEZ/IVA, Symdeko# The study aimed to assess real-world longitudinal changes in FEV1 reduction, BMI, and annual duration of intravenous antibiotic treatment in people with CF homozygous for F508del up to 3 years after the introduction of dual CFTR-modulating therapy LUM/IVA and TEZ/IVA, using NCFR data from 2010 to 2019. A total of 401 CF patients with the F508del/F508del mutation were included.
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14.04.2025
To your attention "Guidelines for the initiation and termination of cystic fibrosis therapy using modulators of the cystic fibrosis transmembrane conductance regulator - CFTR", which was presented by doctors from the Center for Orphan Diseases and Gene Therapy of the National Research and Development Center "Okhmatdyt" of the Ministry of Health of Ukraine. Important information in Ukrainian for everyone who starts modulatory therapy, as well as clear recommendations on monitoring, examinations and tests of patients during the first year of treatment, adverse reactions and criteria for stopping treatment.
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27.03.2025
Vertex has introduced Alyftrek, a new drug that is already approved in the US for children aged 6 and over. The main advantage is the expanded spectrum of mutations, which includes not only those that are amenable to treatment with Trikafta, but also for some mutations of the 3rd, 4th and 5th classes.
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17.03.2025
Mutational dysfunction of the CFTR protein results in multisystem disease, but the greatest morbidity is typically seen in the respiratory tract, where mucus clogs the airways, leading to inflammation and chronic infection and ultimately respiratory failure. CFTR modulator therapy targets the underlying protein defect that leads to lung disease in CF. The extent to which these treatments can reduce susceptibility to chronic lung infections remains to be seen.
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13.03.2025
On March 16, 2025, the wording of the Order of the Ministry of Health of Ukraine No. 406 (Appendix to the Order of the Ministry of Health of Ukraine dated March 30, 2023 No. 598) made changes to the list of reference centers for rare (orphan) diseases that have Cystic Fibrosis among their areas of treatment.
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07.03.2025
Cystic fibrosis is a genetic disease that affects the respiratory and digestive systems. However, due to nighttime coughing, shortness of breath and reduced oxygenation, patients often have sleep disturbances and, as a result, increased fatigue during the day.
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24.02.2025
Starting from 2023 and up to now, most patients in Ukraine with the F508del mutation and aged 6 years and over have already started therapy with a gene corrector. The uniqueness of "Tricaft" lies in the fact that the drug normalizes the function of chloride channels of all cells that produce mucus, thereby eliminating the symptoms of cystic fibrosis. And today we have another piece of good news! We, as Ukraine, have received permission to expand the list of dispensing of the drug "Tricaft" for Ukrainians, which is available in Ukraine. The permission is based on the successful completion of clinical trials among patients with rare mutations in European countries.
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07.02.2025
CFTR modulators are small molecules that enhance or restore epithelial transport of chloride and bicarbonate ions in people with certain mutations that cause CF. Currently licensed modulators include ivacaftor, lumacaftor, tezacaftor, elexaftor, and combinations thereof. Patients taking modulators experience relief, with minimal coughing, reduced mucus production, and difficulty obtaining sputum from the lower lung for pathogenic bacteria. This makes it difficult to detect P.aeruginosa and other dangerous pathogens, both in chronic disease and during exacerbations. Inhaled antibiotics are known to be the standard of care for chronic P.aeruginosa infection and are suppressive therapy for other infections, such as Achromobacter and Stenotrophomonas, in patients with cystic fibrosis.
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03.01.2025
Dear patients and parents, we would like to remind you that a project to identify rare mutations (sequencing of the cystic fibrosis gene by the NGS method using the CFTR panel) is underway, which is free for Ukrainians. All detailed information can be obtained by calling the Center for Orphan Diseases and Gene Therapy +380 (63) 383 04 21 (mobile, Viber, Telegram). In the article below, our doctors-scientists from the Okhmatdyt National Research Center explain in detail for whom and why it is so important to take advantage of the opportunity to undergo the examination: “Cysic fibrosis (CF) is a genetically determined disease caused by pathogenic variants of rearrangements in the transmembrane regulatory protein CFTR gene, which leads to dysfunction of the exocrine glands and primarily affects the respiratory system and digestive tract. In Ukraine, cystic fibrosis occurs with a frequency of approximately 1:2200 newborns.
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30.12.2024
To clear the bronchial tree of viscous sputum in cystic fibrosis, physical methods of sputum evacuation are necessarily used. First of all, for newborns and children under 3 years of age, after inhalation procedures, it is recommended to perform drainage massages of the chest. After three years, you can use breathing exercises, drainage massages on a fitball and jumping on a trampoline. These procedures should be performed twice, three times a day, after each inhalation procedure, preferably with a mucolytic drug...
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17.12.2024
The EU Regulation on Healthcare Assessment (HAA) will enter into force in Europe on 12 January 2025, marking a watershed in the way health technologies and medicines are currently assessed in Europe. This new regulatory framework is designed to revolutionise the assessment of new medicines and healthcare technologies, improve collaboration, efficiency and inclusiveness, with promising implications for orphan communities. By streamlining processes, the Regulation can help speed up access to essential treatments in EU Member States.
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02.12.2024
Another charitable tranche of medicines from the "Atemspende e.V" association was received by the Lviv Regional Center for the provision of medical care to patients with cystic fibrosis. On November 9, Mr. Wolfgang Rackebrandt personally delivered the medicines, namely antibiotics, vitamins in syrup and other medicines to the patients. A number of organizers and benefactors were involved in this event: www.muko.info, Roberto von Alveolus Biomedical, Thomas Kasper, Niklas Vogel, Krystyna Poplavska, Wolfang Rackebrandt and other Deutschland people.
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24.08.2024
Today we want to tell about an incredibly strong girl with cystic fibrosis – Sophie Grace Holmes, who lives in England. The other day, she was recognized as the world record holder among women with cystic fibrosis, who covered the marathon distance every day, for 36 days in a row. This event took place from April 21 to May 26, 2024.
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10.07.2024
07/09/2024 is a day of mourning. But for Ukraine, every day is a day of mourning! Every day we lose our people, children, defenders... On July 8, 2024, the aggressor country Russia once again launched a mass missile attack on our country. One of the rockets hit the country's main children's hospital, Okhmatdyt, Kyiv! The toxicology building, where children were receiving dialysis, was hit, and intensive care, operating, oncology, and other departments were also destroyed. Emergency rescue operations lasted more than a day, as a result of the shelling, 2 people died, more than 50 were injured.
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05.07.2024
Two CF patients have already started taking Choices' newest medicine. In this annual brochure from the Hit-CF team, you can familiarize yourself with the information. Patients whose organs are stored in European laboratories also have a chance to get into the trials!
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27.06.2024
From October 24 to 26, 2024, as part of the extension of the "Twinning" project, a conference will be held in Southern Europe in Cluj-Napoca, Romania on cystic fibrosis. The conference is organized jointly with CF Europe, ECFS and the Romanian participating organizations: Association "Together for Patrick" and Asociata de Fibroza Chistica Din, Romania. The ECFS/CFE Twinning project aims to build friendship and partnership between CF communities in different countries. In 2020, nine centers were established as part of the project. CF centers are united to improve the care of cystic fibrosis patients across Europe. The principle of cooperation is as follows: a cystic fibrosis center, which is well-established and has many years of outstanding experience in the treatment and care of patients, becomes a "mentor center" for a "protégé center" that seeks to improve clinical outcomes through guidance, consultation and collaboration.
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17.06.2024
On October 11, 2021, by Order No. 1235 of the Cabinet of Ministers of Ukraine (amended to Order No. 377 of the Cabinet of Ministers of Ukraine dated April 28, 2021), the Concept of the Development of the System of Providing Medical Assistance to Citizens Developing Rare (Orphan) Diseases was approved. for 2021-2026. . It is aimed at solving the main problematic issues in the system of providing medical care to patients who need treatment for rare diseases by implementing a plan of measures aimed at fulfilling specific tasks. One of the tasks of the concept is "Approving the list of reference centers for rare (orphan) diseases and making changes to it if necessary." The Order of the Ministry of Health No. 864 dated May 21, 2024 (amended to the Order No. 598 of the Ministry of Health dated March 30, 2023) approved the updated List of Reference Centers for Rare (Orphan) Diseases.
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01.06.2024
"Vertex Pharmaceuticals" (Vertex Pharmaceuticals) has developed a new scheme for the treatment of cystic fibrosis - more effective and convenient to use than the combined drug "Trikafta" / "Caftrio", which is now considered the most effective drug for the treatment of patients. The new triple combination consists of drugs: vanzacaftor (vanzacaftor), tezacaftor (tezacaftor) and deutivacaftor (deutivacaftor).
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24.05.2024
May 2024 is full of news for cystic fibrosis patients and families. The unique Simeox medical device, designed to alleviate the condition of children suffering from cystic fibrosis and other lung diseases, was handed over to the department of the Orphan Center of the capital's Children's Medical Center "Okhmadit" the other day.
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03.04.2024
Today is a special date for our wards and for us. Since 2008, February 29 has been the International Day of Rare (Orphan) Diseases. The main goal of this day is to increase society's understanding of rare diseases, their impact on the lives of patients in all aspects, to identify rare diseases as one of the priorities for the health care system, to attract attention to the development of research necessary for safe and effective supportive therapy and treatment of rare diseases...
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