September 23, 2022
In our news update a few months ago we told you about cystic fibrosis, a rare disease that is one of the areas of expertise of our experienced medical and scientific writing team. In particular, we have already helped our clients with a range of projects on this topic, including:
- Congress reports of the international North American cystic fibrosis research (NACFC) held in the US
- Writing a scientific article from a day of exchanges between clinicians and biologists
- Translation of management and follow-up recommendations
- Creation of referenced slideshows.
The “Virades de l’espoir”, walks of hope to help in the fight against cystic fibrosis organized by the association Vaincre la Mucoviscidose in France, provide a timely opportunity to focus on this disease. Similar events take place in many countries around the world, including the Great Strides™ walking challenges organized by the Cystic Fibrosis Trust in the UK and the Cystic Fibrosis Foundation in the US.
2002-2022: 20 years of major progress
The year 2022 marks 20 years of major advances in the diagnosis and management of cystic fibrosis in France and around the world:
These measures have made it possible to improve the prognosis of this disease, allowing early diagnosis and multidisciplinary management organized within the CRCMs from the first weeks of life thanks to neonatal screening. The benefits for patients have been observed at all levels of care, with a favorable cost-benefit ratio:
- improved respiratory and nutritional prognoses
- increased survival
- reduction in the burden of treatments.
More recently, as we mentioned in our news update, innovative medicines, such as the triple therapy elexacaftor-tezacaftor-ivacaftor (Kaftrio®), have also led to a rapid improvement in the health status and quality of life of eligible patients. This treatment was first administered to a very small number of patients in France, thanks to an “autorisation temporaire d’utilisation nominative”, ATUn* [nominative temporary authorization for use]. However, marketing authorization (MA), extension of the indication, and inclusion of Kaftrio® in the compassionate prescription framework of the French National Agency for the Safety of Medicines and Health Products (ANSM), has gradually led to a larger number of patients being able to benefit from this treatment.
Further progress is needed
Despite these important advances, many patients are still missing out on innovative treatments. In addition, there are currently no curative treatments for patients with cystic fibrosis. Thus, further efforts are needed to continue to combat this disease.
“Virades de l’espoir”: Walks of Hope to raise awareness and funds
The “Virades de l’espoir”, created in 1985, help raise awareness of the fight against cystic fibrosis and raise funds to improve the lives of patients with the disease, most notably by funding research into the development of new treatments.
On or around Sunday, September 25 2022 people all over France will participate in the “Virades de l’espoir” and/or take on online challenges to support this fight. Find the complete program here: https://virades.vaincrelamuco.org/
*Name prior to the reform of derogation-based access to medicine schemes which came into force on July 1, 2021 in France.