Newly Diagnosed
So, what do you do now?
It is imperative that you seek out medical guidance from a physician knowledgeable about the disease to confirm diagnosis and move forward on treatment.
MLD is an ultra-rare condition, you should seek a referral to a specialist with expertise in myelin disorders and leukodystrophies as quickly as possible. If you contact Cure MLD, we will have someone respond to your request within two business days. Currently, we can only conduct such meetings in English.
So, what do you do now?
Get to a specialist
There are only a few hospitals in the world with dedicated MLD specialists. Time matters — the earlier treatment begins, the better the outcomes. Find a care center near you.
For US families
Review your insurance
Once an MLD diagnosis is confirmed, review your health insurance immediately and contact your state’s Department of Welfare to apply for Medicaid. See our Medicaid FAQ.
Understand your options
Learn about treatment
Some children may be eligible for gene therapy (Lenmeldy™) or stem cell transplant. Eligibility depends on the form of MLD and whether symptoms have appeared. Learn about treatment options.
You are not alone
Connect with our community
Sign up below and a member of the Cure MLD team will reach out within two business days. We also host parent support groups led by trained professionals. Sign up here.
Your journey
What to expect after a diagnosis
Every MLD family’s path is different, but here is a general guide to the steps most families navigate after receiving a diagnosis.
Confirm the diagnosis
MLD diagnosis involves brain MRI, blood and urine biochemical testing, genetic testing, and neuropsychological testing. If you have not had all four, ask your specialist about completing the full diagnostic picture. Learn about the tests.
Seek a specialist referral
Contact Cure MLD and we will connect you with a leukodystrophy specialist within two business days. You can also find a list of dedicated care centers on our Find Care page.
Understand treatment eligibility
For children who are pre-symptomatic or early symptomatic, gene therapy (Lenmeldy™) may be an option. Your specialist will evaluate eligibility based on age of onset, symptoms, and genetic profile. Learn who is eligible.
Review insurance and Medicaid
Review your health insurance immediately. For US families, apply for Medicaid through your state’s Department of Welfare. Many hospitals have a social worker who can help. Read our full Medicaid guidance.
Find community and support
Review your health insurance immediately. For US families, apply for Medicaid through your state’s Department of Welfare. Many hospitals have a social worker who can help. Read our full Medicaid guidance.
Would you like to meet other families impacted by MLD?
To learn more about our support groups and resources, sign up here and a representative of Cure MLD will reach out to you in two business days. We host a support groups for families hosted by trained professionals here to support you and your family. Sign up to learn more!
