Gene Therapy to get a treatment for Wolfram Syndrome Searching for a cure for a rare genetic disease Donate More Info
what we are doing

A small group of families and the UAB come together in a pioneering project, which is currently the only one the world.

We are a small association of families affected by a serious neurodegenerative genetic disease: Wolfram Syndrome (also called DIDMOAD).

We are promoting a huge project, with our own resources and with the help of many people like you.

A state-of-art gene therapy research project unique in the world with the aim of getting treatment for this illness.

Gene Therapy

Searching for a cure for a rare genetic disease: Wolfram Syndrome
5.890€ Raised: 180.000€ Goal:
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Help us find a cure.

2021
Fundraising
2022
Research
2024
Request for the authorization to the EMA & FDA.

The research

A STATE-OF-ART PROJECT

This project, based on gene therapy, will be carried out in Spain by the UAB (a spanish University based on Barcelona) and consists of three phases:

  • Preclinical proof of concept in in vitro cells.
  • Preclinical proof of concept in animal model
  • Request for the authorization of designation of Orphan Medication to the EMA & FDA.
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Dr. Assumpció Bosch

UAB Professor

Dr. Miguel Chillon

ICREA Researcher

Research team

The project will be carried out by two high-level Spanish researchers with more than 25 years of experience in gene therapy.

This same research team currently has similar projects underway for other diseases in very advanced stages of research.

How can I collaborate?

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IBAN ES30 3058 0979 9127 2001 2933

Swift CCRIES2AXXX

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Companies collaborating with us

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DC Hoteles

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Roimaiori

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