{"id":3357,"date":"2026-08-28T10:32:49","date_gmt":"2026-08-28T08:32:49","guid":{"rendered":"https:\/\/moirefoundation.nl\/?page_id=3357"},"modified":"2026-09-09T17:33:38","modified_gmt":"2026-09-09T15:33:38","slug":"support-us","status":"publish","type":"page","link":"https:\/\/moirefoundation.nl\/en\/support-us\/","title":{"rendered":"Support us"},"content":{"rendered":"\t\t
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Support us<\/h1>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t
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Together, we can bring a treatment for UGDH syndrome closer<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Gene therapy is advancing rapidly. New technologies are making it increasingly possible to conduct targeted research into treatments for rare genetic disorders.<\/p>

For UGDH syndrome, the genetic cause is known. This provides a clear target for the development of a gene therapy approach. The Moir\u00e9 Foundation has therefore established a research program together with an international research team to further develop this approach and take the first steps towards a potential treatment.<\/p>

Within this program, the necessary expertise has been brought together, the research pathway has been developed, and the different stages of development have been defined.<\/p>

The next step is to carry out this research. Targeted funding is needed to make this possible.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Help start the first phase of research<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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The first phase focuses on producing the gene therapy on a small scale and the efficacy in cells. A dedicated research budget is needed to enable the researchers involved to begin this work.<\/p>

Phase 1 \u2013 Preclinical research<\/b><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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\n\t\t\t\t\t\t\t\t\n\t\t\t\tFunding goal: \u20ac75.000\t\t\t<\/span>\n\t\t\n\t\t
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Once the required funding for this phase is available, the research program can begin.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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\n\t\t\t\t\t\n\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\tMake phase 1 possible<\/span>\n\t\t\t\t\t<\/span>\n\t\t\t\t\t<\/a>\n\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Targeted funding for each research phase<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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The research program consists of several consecutive research phases. Each phase has a clear objective and requires specific research activities and corresponding funding.<\/p>

The Moir\u00e9 Foundation has deliberately chosen a phased approach. For each research phase, the required budget is determined, making it clear what resources are needed to enable the next step in the research program.<\/p>

By securing targeted funding for each phase, results can be carefully evaluated and available resources can be used effectively. This approach ensures that the research program is built up in a responsible manner and that each research phase contributes to the development of a potential treatment for UGDH syndrome.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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The four research phases and the funding target for each phase, from the initial preclinical research to a potential first treatment in patients.<\/figcaption>\n\t\t\t\t\t\t\t\t\t\t<\/figure>\n\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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Why this research needs funding<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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UGDH syndrome is an extremely rare condition, meaning that there is limited commercial incentive to develop a treatment. At the same time, gene therapy is advancing rapidly. These developments are creating new opportunities to investigate targeted treatments for even the rarest genetic disorders.<\/p>

For ultra-rare conditions, patients and their families can play an important role in initiating research. As parents of a child with UGDH syndrome, we took the initiative to help start this research. This initiative led to the establishment of the Moir\u00e9 Foundation, which is committed to the development of a gene therapy for UGDH syndrome.<\/p>

No commercial funding is available for the development of a treatment for UGDH syndrome. The Moir\u00e9 Foundation therefore needs to raise the funding required for the research program.<\/p>

The research program is fully focused on one goal: developing this gene therapy. By funding each research phase step by step, available resources can be used purposefully, while keeping the ultimate goal at the center throughout the process.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Time is critical<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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In UGDH syndrome, where the development of the nervous system is affected, the timing of when a treatment becomes available may have a significant impact on patients\u2019 further development. It is therefore important to advance research into potential treatments n\u00f3<\/span>w. The sooner the research can begin, the sooner the first steps towards a treatment can be taken.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Focused use of funds<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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The Moir\u00e9 Foundation is committed to using available funds as directly as possible for the development of a gene therapy for UGDH syndrome.<\/p>

The funds raised are primarily used to support the scientific research within the gene therapy program and to fund the different research phases. This includes the activities required to further develop the gene therapy approach and take the next steps towards a potential treatment.<\/p>

The foundation operates on a non-profit basis. Board members do not receive compensation for their work, allowing the foundation to direct as much of its available resources as possible towards achieving its research objectives.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t

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Help make the research possible<\/h4>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t
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With your support, the first research phase of the gene therapy program can begin. This will mark an important next step towards a potential treatment for patients with UGDH syndrome. Even if a financial contribution is not possible, sharing the Moir\u00e9 Foundation\u2019s mission and raising awareness of UGDH syndrome can make a meaningful difference.<\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t

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Support us Together, we can bring a treatment for UGDH syndrome closer Gene therapy is advancing rapidly. New technologies are making it increasingly possible to conduct targeted research into treatments for rare genetic disorders. For UGDH syndrome, the genetic cause is known. This provides a clear target for the development of a gene therapy approach. […]<\/p>\n","protected":false},"author":3,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"inline_featured_image":false,"site-sidebar-layout":"no-sidebar","site-content-layout":"page-builder","ast-site-content-layout":"full-width-container","site-content-style":"unboxed","site-sidebar-style":"unboxed","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"disabled","ast-breadcrumbs-content":"","ast-featured-img":"disabled","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"enabled","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"set","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"_locale":"en_US","_original_post":"urn:uuid:99ecb5c7-073d-4f72-9dae-68d42be02ae6","footnotes":""},"class_list":["post-3357","page","type-page","status-publish","hentry","en-US"],"rttpg_featured_image_url":null,"rttpg_author":{"display_name":"Jannes","author_link":"https:\/\/moirefoundation.nl\/author\/jannes\/"},"rttpg_comment":0,"rttpg_category":null,"rttpg_excerpt":"Support us Together, we can bring a treatment for UGDH syndrome closer Gene therapy is advancing rapidly. New technologies are making it increasingly possible to conduct targeted research into treatments for rare genetic disorders. For UGDH syndrome, the genetic cause is known. This provides a clear target for the development of a gene therapy approach.…","_links":{"self":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3357","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/comments?post=3357"}],"version-history":[{"count":22,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3357\/revisions"}],"predecessor-version":[{"id":3752,"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/pages\/3357\/revisions\/3752"}],"wp:attachment":[{"href":"https:\/\/moirefoundation.nl\/wp-json\/wp\/v2\/media?parent=3357"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}