The mission of the VWM Families Foundation is to support and advance research for Vanishing White Matter disease (VWM).

Finding a Cure

Guanabenz trial

We are one of the primary sponsors of the first ever VWM clinical trial. This trial is being conducted in Amsterdam by Dr. Marjo van der Knaap. The trial has shown promising results and we are currently fundraising so this trial can be extended and the drug can be produced at low cost and made available to families all over the world

Click here for more information about the Guanabenz trial

Gene Therapy Research

Dr. Bonkowsky of the University of Utah, in collaboration with Dr. Allison Bradbury of Nationwide Children’s Hospital is currently researching the potential of gene therapy in treating VWM.

For more on this research click here

VWM Consortium & ULF

The Vanishing White Matter Consortium consists of 9 (pediatric) neurologists from 8 academic medical centers worldwide, all involved in leukodystrophy patient care and research. Together with our partners, we work towards a cure for VWM.

The VWMFF works closely with the VWM Consortium as its patient partner.

For more information see www.vwmconsortium.org

United Leukodystrophy Foundation

The United Leukodystrophy Foundation (ULF), incorporated in 1982, is a non-profit, voluntary health organization dedicated to funding cutting-edge research and to providing patients and their families with disease information and medical referrals. The Foundation is located in DeKalb, Illinois.

The ULF mission is to provide support to the leukodystrophy community and enable platforms to accelerate improving patient quality of life and finding cures.

The ULF Conference is held every year in Chicago, Illinois and is a very valuable resource for VWM families. We highly encourage attending the conference at least once

Additional Projects

In the past we also funded some of Professor Orna-Elroy-Stein's research, which led to identification of a compound that may slow the progression of VWM

We also are supporting the Calico clinical trial and VWM consortium by providing patient input in trial design and outcomes

For more on the Calico trial click here https://www.calicolabs.com/clinical-trial-vanishing-white-matter/

Supporting VWM Families

VWM causes the white matter in a patient’s brain to literally disappear and be replaced with water. With it a patient loses motor control. This results in all patients having severe physical disabilites and will inevitably lose the ability to walk.

Many people are suprised to hear that necessary medical equipment is often not covered by insurance. This can include motorized wheelchairs, feeding chairs, bathing seats, and standers.

In addition, as a child with VWM gets older it becomes impossible for families to include their VWM child in everyday life without disabled access in and out of the home and a wheelchair van to get their child around without having to lift them out of a wheelchairs. These things are not always covered by insurance and families are expected to pay for them out of pocket. The financial burden on top of the stress of a sick child is more than families should have to bear.

We aim to help families by helping fund this necessary equipment and ease their financial burden.

Donate: It is up to the VWM Families Foundation to raise the money to keep these projects going.

We have an ambitious goal of raising $500,000 in the next year. We are so grateful for all the efforts and contributions to date and know with your help we will reach our goal.