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btn__header btn__desktop" href="https://www.vwmconsortium.org/register-as-vanishing-white-matter-patient/">Register</a> <div class="nav-overlay__toggle" id="nav-overlay__toggle"> <div class="nav-overlay__toggle-lines"> <div></div> </div> </div> <div class="overlay-blur"></div> <div class="nav-overlay"> <ul id="nav-overlay__list" class="nav-overlay__list"><li class="menu-item menu-item-type-post_type menu-item-object-page menu-item-38"><a href="https://www.vwmconsortium.org/about-us/">About us</a></li> <li class="menu-item menu-item-type-post_type menu-item-object-page menu-item-30"><a href="https://www.vwmconsortium.org/about-vwm/">For VWM patients</a></li> <li class="menu-item menu-item-type-post_type menu-item-object-page menu-item-31"><a href="https://www.vwmconsortium.org/clinical-trials/">Clinical Trials</a></li> <li class="menu-item menu-item-type-custom menu-item-object-custom menu-item-has-children menu-item-220"><a>Updates</a> <ul class="sub-menu"> <li class="menu-item 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class="content"> <div class="content__inner"> <div class="cb cb__banner-image cbbi " style="background-image: url(https://www.vwmconsortium.org/wp-content/uploads/2021/06/iStock-187272969_medium.jpg);"> <div class="container"> <div class="row"> <div class="col-12"> <div class="cbbi__content"> <h1 class="cbbi__title">The VWM consortium published an expert opinion on therapy trial design</h1> </div> </div> </div> </div> </div> <div class="cb cb__content_rows cbcr pt-l pb-m bg--light-purple"> <div class="container"> <div class="row justify-content-center" data-aos="fade-up" data-aos-delay="100"> <div class="col-12 col-sm-10 col-md-8 mb-m"> <p>With increased insight into Vanishing white matter and potential drugs in early development, it is time to start thinking about clinical trial design. Considering several challenges (such as low numbers of patients, unpredictable disease course, absence of intermediate biomarkers), smart clinical trial design and close collaboration with all stakeholders is key. In the first VWM consortium paper together with patient associations, we have formulated recommendations for enhanced diagnosis, drug trial setup, and patient selection, based on our expert evaluation of molecular, laboratory, and clinical data.</p> <p>This is an important step in the drug development landscape for VWM. Please read our published paper in Neurology Genetics <a href="https://ng.neurology.org/content/8/2/e657">here</a>.</p> <p> </p> </div> </div> </div> </div> <div class="single__related-news bg--light-purple p-l"> <div class="container"> <div class="row"> <div class="col-12"> <div class="cbn__titles"> <div class="h2 cbn__title">More News Articles</div> <a class="btn--round" href="https://www.vwmconsortium.org/news/"> <div class="btn--round-text">All News</div> <div class="btn--round-arrow"></div> </a> </div> </div> </div> <div class="row"> <div class="col-12 col-sm-6 col-md-4"> <a href="https://www.vwmconsortium.org/the-core-protocol-for-phase-2-3-clinical-trials-in-vwm-is-now-published/" class="card card-news"> <div class="card-news__image" style="background-image: url(https://www.vwmconsortium.org/wp-content/uploads/2021/06/Teamwork_medium-640x480.jpg);"> </div> <div class="card-news__content bg--white"> <div class="card-news__btn btn--round"> <div class="btn--round-arrow"></div> </div> <div class="card-news__date">21 augustus 2023</div> <div class="h4 card-news__title">The core protocol for phase 2/3 clinical trial(s) in VWM is now published</div> <div class="card-news__text">The core protocol for phase 2/3 clinical trial(s) in VWM, aimed to serve as a template for industry-sponsored clinical trials in VWM, is now published. </div> </div> </a> </div> <div class="col-12 col-sm-6 col-md-4"> <a href="https://www.vwmconsortium.org/calico-life-sciences-announces-first-participant-dosed-in-phase-1b-clinical-study-evaluating-abbv-cls-7262-for-the-treatment-of-vanishing-white-matter-disease/" class="card card-news"> <div class="card-news__image" style="background-image: url(https://www.vwmconsortium.org/wp-content/uploads/2021/06/Teamwork_medium-640x480.jpg);"> </div> <div class="card-news__content bg--white"> <div class="card-news__btn btn--round"> <div class="btn--round-arrow"></div> </div> <div class="card-news__date">26 april 2023</div> <div class="h4 card-news__title">Calico Life Sciences Announces First Participant Dosed in Phase 1b Clinical Study Evaluating ABBV-CLS-7262 for the Treatment of Vanishing White Matter Disease</div> <div class="card-news__text">Calico Life Sciences Announces First Participant Dosed in Phase 1b Clinical Study Evaluating ABBV-CLS-7262 for the Treatment of Vanishing White Matter Disease</div> </div> </a> </div> <div class="col-12 col-sm-6 col-md-4"> <a href="https://www.vwmconsortium.org/calico-starts-phase-1b-study-evaluating-safety-tolerability-and-pharmacokinetics-of-eif2b-activator/" class="card card-news"> <div class="card-news__image" style="background-image: url(https://www.vwmconsortium.org/wp-content/uploads/2021/06/iStock-1185390850_medium-640x480.jpg);"> </div> <div class="card-news__content bg--white"> <div class="card-news__btn btn--round"> <div class="btn--round-arrow"></div> </div> <div class="card-news__date">26 maart 2023</div> <div class="h4 card-news__title">Calico starts phase 1b study evaluating safety, tolerability and pharmacokinetics of eIF2B activator</div> <div class="card-news__text">Calico starts phase 1b study evaluating safety, tolerability and pharmacokinetics of eIF2B activator. </div> </div> </a> </div> </div> </div> </div> </div> </main> <footer class="footer p-l"> <div class="container"> <div class="row"> <div class="col-12 col-sm-6 footer__logo__text"> <div class="footer__logo__img"></div> <p>The Vanishing White Matter consortium consists of 9 (pediatric) neurologists from 8 academic medical centers worldwide, all involved in leukodystrophy patient care and research. The consortium started in 2019 to work on the overarching mission: enabling and accelerating therapeutics for Vanishing White Matter, accessible and affordable for all patients. To help us achieve our mission we interact with various partners, including patient / family / caregiver foundations, (medical) specialists, academic institutes, and biotech and pharma companies.</p> </div> <div class="col-12 col-sm-6 footer__registry"> <div class="footer__registry__content"> <h3 class="footer__registry__titel">VWM patient registry</h3> <div class="footer__registry__text"> <p>The Amsterdam Leukodystrophy Center has developed an online global registry for patients with Vanishing White Matter (VWM). The purpose of the registry is to gain as much insight as possible into the course of the disease, both in terms of clinical features and MRI. This database will play an important role in developing treatment strategies and making decisions about therapeutic trials. Information is needed about as many patients as possible.</p> </div> <a href="https://www.vwmconsortium.org/register-as-vanishing-white-matter-patient/" class="footer__registry__button btn btn--green">Register</a> </div> </div> </div> <div class="row footer__bar justify-content-between"> <div class="col-12 footer__bar-col"> <div class="footer__copyright"> <div class="footer__bar-links">© 2025 <p>Amsterdam UMC • <a href="https://www.amsterdamumc.org/research/disclaimer.htm" target="_blank" rel="noopener">Disclaimer</a> • <a href="https://www.amsterdamumc.org/research/general-privacy-statement.htm" target="_blank" rel="noopener">Privacy Policy</a></p> </div> </div> <a href="https://www.stoerebinken.nl/" class="footer__made-by" target="_blank">Website: Stoere Binken</a> </div> </div> </div> </footer> <script type="text/javascript" src="https://unpkg.com/aos@next/dist/aos.js?ver=6.7.2" id="aos-js"></script> <script type="text/javascript" src="https://www.vwmconsortium.org/wp-includes/js/imagesloaded.min.js?ver=5.0.0" id="imagesloaded-js"></script> <script type="text/javascript" src="https://www.vwmconsortium.org/wp-includes/js/masonry.min.js?ver=4.2.2" id="masonry-js"></script> </body> </html>