Frequently Asked Questions
Is there a cure? What treatments are available?
While leukodystrophy does not have a cure (yet!) there are treatments available. The type of leukodystrophy diagnosed will dictate what treatment options to pursue. Some leukodystrophies only have supportive treatments – which are meant to relieve symptoms. Supportive treatments do not halt or slow the progress of the disease. You will need to consult a licensed medical professional who has access to your complete medical history to discuss your treatment plan.
I have a relative that was diagnosed with leukodystrophy. Do I have leukodystrophy too?
We advocate for families to speak with genetic counselors to determine whether testing is appropriate for family members, based on the type of leukodystrophy that is diagnosed. In some cases, only certain family members would need to be tested and a genetic counselor would be able to answer those questions.
For example – some leukodystrophies are carried only on the X chromosome. If a male with the affected X chromosome has a daughter with an unaffected female, the daughter will be a carrier of the mutated gene on the X chromosome passed to her from her affected father. If the affected male and unaffected female has a son, the son will not be affected and will not carry the mutated gene, as the father gave his son an unaffected Y chromosome. Your primary care physician should be able to provide a referral to a genetic counselor in your area. The ULF can assist as well.
Invitae offers genetic testing to families from the United States, Canada, and Australia, who are at risk of leukodystrophy at no cost! You can find more information here: https://www.invitae.com/en/leukodystrophies/
Why isn’t there a cure yet? The leukodystrophies have been around for decades!
Research for a cure is moving as fast as it can! In the meantime, researchers from all over the world are also looking into improving therapies to halt or at least slow the disease progression. Scientists need to study the disease for many years to determine exactly what is happening to a person’s body as they fight the disease. Understanding how the disease itself works is the only way to find an effective cure. The ULF stresses that even “failed” studies provide answers to the scientific community, because now the researchers are aware what roads are dead ends and can focus on more promising avenues. Quality and length of life for leukodystrophy patients are slowly but surely improving with each passing year, as research continues to advance. The ULF is dedicated to making sure that the projects funded by ULF grants are some of the most promising projects in progress today.
What can I expect, as a patient or as a caregiver, in terms of symptoms or how quickly the disease will progress?
Each person with a leukodystrophy diagnosis will face very different obstacles. The first question is determining which type of leukodystrophy is being diagnosed. Each type has its own distinct characteristics in terms of symptoms, its onset, and speed of progression. The overall health of the affected individual must also be factored in. The patient’s primary care team is the best resource to consult with specific questions such as this – there are many factors to consider.
What does the ULF do?
We are a resource for families affected by leukodystrophy to help them find the assistance they need to better their quality of life, and we fund scientific research through our annual grant cycle program. Learn more.
Can I send you MRI or test results for a second opinion?
We ask that you do not send us any medically sensitive information. The ULF does not have any medical professionals on staff to review this information. Due to privacy restrictions, the ULF cannot pass along patient information to medical professionals. What we can do is help you find a physician to speak with, and you can send your information directly to them.
Are there leukodystrophy support groups that meet in my hometown or state?
The annual ULF Family Conference is the next best thing to a support group. Other Patient Advocacy Organizations host conferences as well, which are included in the ULF Calendar of Events. The ULF is unaware of any physical leukodystrophy support groups that meet on a regular basis. You could inquire with your local social service foundations in your area to see if there are any support groups that could be of interest to you. Hospitals or mental health facilities will occasionally host support groups for chronic illness or terminal disease patients. While these groups are not specific to leukodystrophy, they may give you the sense of community that you are looking for in your immediate area. If you have a Facebook profile, you could search for leukodystrophy groups to join, including general leukodystrophy and your specific type diagnosis if it is known.
I need financial assistance. Does the ULF have any financial relief programs?
We do offer scholarships to help families in financial need to attend our annual Conference. Find more information on the scholarship program.
Can I donate to a specific type of leukodystrophy research?
Yes! Every year, we take a look at how much has been donated to our general research fund and our specific type funds which guides our MSAB on how to award the funds. Learn more about our grant cycle.