CURE for Children with Cockayne Syndrome – type B

  • €1,805.00
    raised of €500,000.00 goal goal
0% Funded
23 Donors
Help this ongoing fundraising campaign by making a donation and spreading the word.
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Donate for development of gene therapy for children with deadly disease Cockayne syndrome – type B.

Cockayne syndrome – type B (CSB) is a very rare, terrible and deadly genetic disease. Currently there is NO cure or treatment for CSB, leaving children and their families in a desperate search for hope. The only hope for children with CSB is development of gene therapy which would be a treatment for this disease.

In non-profit charity organization Viljem Julijan Association for Children with Rare Diseases we have in cooperation with parents of children with CSB partnered with scientists which are now developing gene therapy for CSB. Development of gene therapy costs 2.000.000€. To date we have already raised 1.431.000€. We are now at a critical juncture, with 500.000€ still needed to reach our target. Your support can bring us closer to a treatment that has the potential to save lives of children.

Time Is of the Essence: We Need Your Support Now!

At the Viljem Julijan Association, we are fueled by a mission of love and desperation. Our quest for a gene therapy was sparked by the brave heart of a 5-year-old girl from Slovenia, Karolina, whose life hangs in the balance as she faces the challenges of Cockayne Syndrome - Type B. Inspired by Karolina's fight, and united with her family's dreams of saving her, we've embarked on a brave journey towards developing a lifesaving gene therapy.

More about Cockayne syndrome - type B

CSB a progressively devastating disease, characterized by early severe progression of symptoms which display multiorgan dysfunction. It causes premature aging (progeria), intellectual disability, photosensitivity, and moderate to severe learning delay. Individuals with CSB may also have photosensitivity, hearing loss, vision problems, dental abnormalities, and skeletal abnormalities. The average lifespan of children with CSB in 12 years.

The disease is caused by mutations in the ERCC6 gene. This gene provides instructions for making a protein called DNA excision repair protein or CS group B protein (CSB) This protein plays a role in repairing damaged DNA.

Development of gene therapy for Cockayne syndrome - type B

Our research teams in USA lead by dr. Christina Pacak from University of Minnesota and in Portugal lead by dr. Clévio Nóbrega from Algarve Biomedical Research Institute are now raicing towards development of gene therapy for CSB.


Your Contribution Can Make a World of Difference

Imagine a world where no child has to suffer from Cockayne Syndrome - Type B ever again. By donating to our campaign, you're not just giving funds; you're giving hope, time, and the promise of a future to children like Karolina and many others. Your generosity will directly support the development of gene therapy, marking you as a crucial ally in this fight against time.

Together, We Can Turn the Tide Against CSB

This is a call to action, a plea for compassion, and a challenge to make a difference. Let's unite in this fight to conquer Cockayne Syndrome - Type B. Your donation today is more than a gift; it's a lifeline for those in the grips of this devastating disease. Join us on this mission of hope and healing. Together, we can save lives and herald a new dawn for children battling CSB.

Don't Wait – Act Now!

Your support can change the course of history for these children and their families. Help us reach our goal of 2.000.000€. Every contribution, no matter the size, brings us closer to a cure. Be the change. Be the hope. Donate now and help us save precious lives from the clutches of Cockayne Syndrome - Type B.

Together, we can make the impossible, possible. Join us. Save a life.

About Viljem Julijan Association for Children with Rare Diseases

Viljem Julijan Association for Children with Rare Diseases is a registered non-profit charity organization from Slovenia, European Union. It was established in 2018 by Gregor Bezenšek (musician SoulGreg Artist) and his wife Nina, parents of a boy Viljem Julijan who in 2019 died from rare genetic disease GM1 gangliosidosis.

Viljem Julijan Association is a beacon of hope and support for children and their families who are facing the challenging journey of rare diseases. Founded in loving memory of Viljem Julijan, a brave little soul who battled a rare disease, this association has grown into a vital community resource, dedicated to improving the quality of life for children afflicted with rare conditions and their families.

Our mission is to provide comprehensive support, including emotional, informational, and financial assistance, to families affected by rare diseases. We understand the profound impact these conditions can have on the entire family, which is why our approach is holistic and family-centered. Through our efforts, we aim to raise awareness about rare diseases, advocate for improved medical and social care, and fund research initiatives that promise better treatments and, ultimately, cures.

The Viljem Julijan Association is more than just an organization; it's a community where families find understanding, strength, and hope. Our initiatives include organizing family retreats, educational workshops, and social events that offer respite and joy to children and their loved ones. Furthermore, we tirelessly work to bridge the gap between medical professionals and families, ensuring that the voices of those affected by rare diseases are heard and considered in the healthcare landscape.

As we launch our fundraising campaign for development of gene therapy for Cockayne syndrome – type B, we invite you to join us in making a significant difference in the lives of these extraordinary children and their families. Your contribution will directly support research work done by scientist in USA and Portugal.

Stand with us in this noble cause, for every child deserves the chance to thrive.

Fundraising Team

  • Marjeta Kobal
  •  
  • Social media manager

Donors

  • Katarina Sablicova
  • Donated on Mar 31, 2024
€50.00
  • Neva Groselj
  • Donated on Mar 30, 2024
€50.00
  • Geana Dyer
  • Donated on Mar 25, 2024
  • From: Uncle Jim and Joyce For: Mr. Ronin and his CS friends ❤️

€92.00

No updates for this campaign just yet

Donors & Comments

23 donors
  • Katarina Sablicova
  • Donated on Mar 31, 2024
€50.00
  • Neva Groselj
  • Donated on Mar 30, 2024
€50.00
  • Geana Dyer
  • Donated on Mar 25, 2024
  • From: Uncle Jim and Joyce For: Mr. Ronin and his CS friends ❤️

€92.00
  • Ian Brassard
  • Donated on Mar 25, 2024
€100.00
  • Lorrie Cartier
  • Donated on Mar 25, 2024
  • ❤️❤️❤️🙌🙌!!

€50.00
  • Geana Dyer
  • Donated on Mar 24, 2024
  • From: Uncle Bob and Aunt Sue Brassard For: Ronin ❤️

€92.00
  • Geana Dyer
  • Donated on Mar 24, 2024
  • From: Brandie and Matt Hammons To: Ronin, our Ray of Sunshine ☀️ i

€92.00
  • Tom & Vikki Davison
  • Donated on Mar 24, 2024
€100.00
  • Josh and Christina Davison
  • Donated on Mar 24, 2024
  • ❤️

€100.00
  • Jerry & Theresa Davison
  • Donated on Mar 23, 2024
€100.00
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Followers

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Jamie Davison
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Marjeta Kobal
€1,805.00
raised of €500,000.00 goal
0% Funded
23 Donors

Help this ongoing fundraising campaign by making a donation and spreading the word.

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