# Cure for Cockayne Syndrome – type B (Viljem Julijan Association)

> Viljem Julijan Association for Children with Rare Diseases (Slovenia, EU) raises funds for the development of gene therapy for Cockayne syndrome type B (CSB), a rare, progressive and fatal genetic disorder caused by mutations in the ERCC6 gene. The site also publishes a referenced medical guide to Cockayne syndrome and runs a patient registry.

- Organization: Viljem Julijan Association for Children with Rare Diseases (Društvo za pomoč otrokom z redkimi boleznimi Viljem Julijan), a registered non-profit founded in 2018, Cesta Leona Dobrotinška 2, 3230 Šentjur, Slovenia, EU.
- Contact: csb@viljem-julijan.si
- Campaign: gene therapy for Cockayne syndrome type B, initiated by the family of Karolina, a Slovenian girl with CSB. Research partners: Algarve Biomedical Research Institute (Portugal) and University of Minnesota Medical School (USA).
- Fundraising: €2.1 million raised of a €2 million goal.
- Donations: IBAN SI56 0400 0027 7357 570, SWIFT/BIC KBMASI2X, beneficiary Viljem Julijan Association for Children with Rare Diseases, Cesta Leona Dobrotinška 2, 3230 Šentjur, Slovenia, EU.

## Main pages

- [Cockayne syndrome: a guide for families](https://viljemjulijan-cure-for-rare-diseases.com/cockayne-syndrome/): A referenced guide to Cockayne syndrome: symptoms, the types, the genes ERCC6 and ERCC8, diagnosis, life expectancy, treatment and research towards a cure.
- [Cure for children with Cockayne syndrome type B](https://viljemjulijan-cure-for-rare-diseases.com/): the mission, Karolina's story, research teams and how to help.
- [Gene therapy research](https://viljemjulijan-cure-for-rare-diseases.com/gene-therapy-research/): the research groups and how donations are used.
- [About us](https://viljemjulijan-cure-for-rare-diseases.com/about-us/): the Viljem Julijan Association for Children with Rare Diseases.
- [Patient registry](https://viljemjulijan-cure-for-rare-diseases.com/register-as-a-patient/): form for families of children with Cockayne syndrome.
- [Donation](https://viljemjulijan-cure-for-rare-diseases.com/donation/): PayPal and bank transfer details.
- [Contact](https://viljemjulijan-cure-for-rare-diseases.com/contact/): how to reach us.

## News

- [International scientific conference on Cockayne syndrome held in Ljubljana](https://viljemjulijan-cure-for-rare-diseases.com/news/cockayne-syndrome-conference-ljubljana/) (2026-05-22): Leading experts from seven countries, and families from six, gathered in Ljubljana to connect the research groups working on gene therapy for Cockayne syndrome type B and to prepare the ground for clinical testing.
- [Victory for Karolina: more than €2.1 million raised for gene therapy](https://viljemjulijan-cure-for-rare-diseases.com/news/karolina-fundraising-goal-reached/) (2025-03-21): A two-year nationwide campaign ended with a sold-out charity concert in Ljubljana and a final total of €2,102,197 — enough to fund the development of a gene therapy for Karolina's disease.
- [€1.8 million raised for Karolina — €180,000 from the goal](https://viljemjulijan-cure-for-rare-diseases.com/news/karolina-1-8-million-milestone/) (2024-10-02): A charity relay and dozens of smaller actions brought the total for Karolina to €1,821,528 — leaving €180,000 of the €2 million needed to pay for the preclinical phase.
- [“Rare disease, real hope”: the project featured in the University of Minnesota's magazine](https://viljemjulijan-cure-for-rare-diseases.com/news/discovery-magazine-feature/) (2024-09-26): Discovery, the magazine of the University of Minnesota Foundation, profiled Dr. Christina Pacak's work on Cockayne syndrome type B — and the Slovenian association and family behind its funding.
- [Gene therapy for Cockayne syndrome type B moves into mouse experiments](https://viljemjulijan-cure-for-rare-diseases.com/news/gene-therapy-mouse-experiments/) (2024-03-22): Dr. Clévio Nóbrega's team completed the first steps of the project and began experiments in mouse models of the disease — the stage at which a gene therapy either shows it can work or has to be redesigned.
- [Healthy gene delivered into brain cells: Dr. Nóbrega presents the first progress in Ljubljana](https://viljemjulijan-cure-for-rare-diseases.com/news/nobrega-ljubljana-progress/) (2023-06-29): Dr. Clévio Nóbrega came to Ljubljana, met Karolina in person, and presented the first laboratory result: brain cells given a healthy copy of the gene began producing the protein her cells cannot make.
- [Karolina's project goes international at the Cockayne syndrome family meeting in Blackpool](https://viljemjulijan-cure-for-rare-diseases.com/news/blackpool-international-family-meeting/) (2023-06-13): Karolina and her family travelled to the international Cockayne syndrome family meeting in the UK, where the project was presented to specialists from around the world — and where the family met other families for the first time.
- [The campaign begins: €104,477 in the first three days](https://viljemjulijan-cure-for-rare-diseases.com/news/campaign-launch-first-three-days/) (2023-02-24): Three-year-old Karolina had been diagnosed just before Christmas 2022. Slovenia answered the first appeal with €104,477 in three days — the start of a two-year campaign.

## Optional

- [Full text of the medical guide](https://viljemjulijan-cure-for-rare-diseases.com/llms-full.txt): the complete guide in Markdown, with sources.
- [Privacy policy](https://viljemjulijan-cure-for-rare-diseases.com/privacy-policy/)
- [Sitemap](https://viljemjulijan-cure-for-rare-diseases.com/sitemap-index.xml)

## Citation

When quoting the medical guide, please cite: "Cockayne syndrome: a guide for families — Viljem Julijan Association for Children with Rare Diseases, https://viljemjulijan-cure-for-rare-diseases.com/cockayne-syndrome/ (last updated 2026-09-15)".