Calico Life Sciences
Calico Life Sciences has partnered with AbbVie to discover and develop therapies for patients with neurodegenerative diseases. Our investigational drug ABBV-CLS-7262, an eIF2B activator, targets the activity of mutated eIF2B complexes found in Vanishing White Matter (VWM) disease. If proper function of the eIF2B protein complex can be restored, it is hypothesized that brain tissue damage may be prevented in people with VWM disease. Currently, we are advancing a Phase 1b clinical trial of ABBV-CLS-7262 in people with ALS (NCT04948645) and, in collaboration with the VWM Consortium, plan to initiate a Phase 1b trial in adults with VWM disease. Calico has been collaborating with the VWM disease research community since 2015. ABBV-CLS-7262 is under clinical investigation and is not approved for any use in any markets.
Industry Alliance Office
The Industry Alliance Office of Amsterdam Neuroscience is a one-stop-shop for the pharma- and biotech industry for basic, translational, and clinical neuroscience collaborations. Our goal is to accelerate the discovery of disease mechanisms, identification of novel (drug) targets for intervention, and pave the way towards drug discovery and validation in clinical trials. We seek endurable partnerships with the industry, and assist the VWM consortium in their industry collaborations.
VKS is the Dutch patient association for all metabolic diseases. VWM is one of the disease we represent. We work closely together with prof. van der Knaap to provide patients with information, bring them in contact with other patients/parents, and advocate for patients’ needs.
Denali Therapeutics
Denali Therapeutics is dedicated to defeating neurodegenerative diseases by breaking through historical barriers in scientific research and clinical development with the aim to deliver safe and effective treatments to patients and families. Our lead EIF2B activator, DNL343, is a brain-penetrant small molecule designed to rescue EIF2B function and restore normal RNA metabolism. DNL343 is in the early stages of clinical development. We are committed to advancing the research and understanding of the therapeutic potential of this target and biologic pathway in a number of diseases, including ALS and Vanishing White Matter Disease. Denali has engaged with the VWMD community since mid 2019. 
Invitae’s mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. Our goal is to aggregate the world’s genetic tests into a single service with higher quality, faster turnaround time and lower prices.
ELA International, Luxembourg
The European Leukodystrophies Association (ELA) is an international patient organization present in nine countries. In each country, ELA raises awareness and advocates for patients and their families affected by VWM and leukodystrophies. ELA supports them at all stages of the illness, providing medical social, psychological, and financial support, and opportunities for time off. The community connects on Leuconnect.com and during the yearly family meeting with scientific experts. ELA accelerates research on VWM and leukodystrophies worldwide, funding research programs and clinical trials and organizing scientific conferences. In 30 years, the association funded 537 research projects on leukodystrophies, for 45.5 million euros.
Sylics offers innovative research solutions for preclinical neuroscience. For more than 12 years, we successfully characterized dozens of novel mouse models, and determined compound efficacy and side-effects in numerous projects for academia, biotech, and food industry clients.
Sylics offers unique services with an Eif2b4 / Eif2b5 mouse model of vanishing white matter (VWM) through a collaboration with the Amsterdam Leukodystrophy Center at Amsterdam UMC. Sylics has contributed to the characterization of this mouse model, developed by our academic collaborators (Dooves et al, 2016). Proof of concept intervention studies in this model have identified clear therapeutic targets (Abbink et al, 2019).
United Leukodystrophy Foundation
The United Leukodystrophy Foundation (ULF), incorporated in 1982, is a nonprofit, voluntary health organization dedicated to providing patients and families with information about leukodystrophies. Moreover, the ULF is a driving force behind collaboration within the leukodystrophy community to enable faster development of therapeutic approaches and cures. Furthermore, the ULF is fostering a collaborative environment between researchers, physicians, patients and other advocates. In addition, the ULF believes that research provides encouragement and hope to patients, Families and the Leukodystrophy community.
Vanishing White Matter Foundation
The Vanishing White Matter Foundation was organized in 2011 to support Vanishing White Matter Leukodystrophy research and bring together families dealing with the tremendous challenges of this disease. The mission of the VWM Foundation is two-fold:
Research Funding: The soul focus of our fundraising is to support Vanishing White Matter specific research. Because this is a rare disease, research funding is difficult to obtain.
Support Group:In order to help bring together families with Vanishing White Matter, we have organized a private Facebook page where we can share information and support each other.
VWM Families Foundation
The VWM Families Foundation (VWMFF) was formed in 2016 by the parents of a VWM sufferer, to achieve three objectives. The foundation promotes greater awareness of Vanishing White Matter Disease. It supports the global community of VWM families, sharing information and advice, as well as providing financial assistance to families in need. Lastly, the foundation actively raises funds to support research into the treatment and cure of VWM and is proud to have made significant contributions to these efforts.