Together to change lives
Contributing to medical research and funding projects dedicated to rare and orphan diseases, particularly those affecting children.
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Contribute to medical research and support initiatives for children affected by rare diseases.
LEARN MORE →Help fund scientific projects aimed at improving our understanding of rare diseases and advancing treatments.
DÉCOUVRIR OUR ACTIONS →Your support helps give researchers the means to pursue essential work for patients and their families.
MAKE A DONATION →Your support will help MJJ Charity Organization contribute to medical research projects dedicated to rare and orphan diseases, particularly those affecting children.
We are currently preparing our fundraising system so that donors can use a secure and transparent way to contribute.
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The online donation form will be activated once our fundraising solution is in place.
With an annual membership fee of €20 per year, you join MJJ Charity Organization and provide lasting support for our mission to contribute to medical research dedicated to rare and orphan diseases, particularly those affecting children.
Each annual membership includes a MJJ Charity Organization membership card, issued after membership approval.
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Our mission is to contribute, within our means, to the funding and support of medical research projects dedicated to rare and orphan diseases, with particular attention to diseases affecting children.
Areas that may benefit from this commitment include epidermolysis bullosa, spinal muscular atrophy, Duchenne muscular dystrophy, certain leukodystrophies, Batten disease, Sanfilippo syndrome and other rare diseases for which research remains essential.
Our objective is to help advance therapeutic solutions, including curative treatments when research makes them possible, without making any advance claim about a medical outcome.
This section will progressively present the projects, events and initiatives of MJJ Charity Organization.
Discover a selection of clothing and accessories in the colors of MJJ Charity Organization. Each item will carry the identity of the organization and help spread awareness of our commitment to research into rare and orphan diseases.
Black T-shirt featuring the organization logo.
Premium black hoodie featuring the organization logo.
The shop is currently being prepared. Secure online payments and ordering will be activated once the sales solution is in place.
The organization’s news, events and projects will be published here.
Research into epidermolysis bullosa (EB) is making major progress. Gene therapies have reached patients, while other approaches are seeking to correct the genetic origin of the disease more precisely.
For certain dystrophic EB forms linked to COL7A1, VYJUVEK delivers a functional copy of the gene directly to wound cells. It has been authorized in the European Union since 2025.
French researchers are studying genetically corrected skin cells from the patient that can be used to create skin grafts. The approach aims to restore local production of collagen VII.
Researchers are exploring more precise correction strategies, including CRISPR, base editing and prime editing. These approaches remain mainly at the preclinical development stage.
Other studies aim to accelerate wound closure, reduce inflammation and improve management of pain, eye involvement and serious complications of some EB forms.
MJJ Charity Organization aims to contribute to and support research into EB and other rare and orphan diseases affecting children. This information is provided for general information and is not medical advice.