After decades of research, the potential for new treatment options is on the horizon for Leukodystrophies. With this comes clinical trials and, hopefully, new and better options for many types of Leukodystrophies.

Clinical Trials

The first place to begin when wanting to learn more about clinical trials for Leukodystrophies is is a resource provided by the U.S. National Library of Medicine and is a database of clinical studies around the world.

This is where you can find the most current information by simply searching your or your loved ones specific disease type.

Learn more about clinical trials through Dr. Joshua Bonkowsky’s presentation at the 2021 Hunter’s Hope Symposium – Clinical Trials 101.

Industry Partners

We are thankful for our Industry Partners who are ushering in this new era for Leukodystrophies. You can learn more about the disease groups they serve and other information below.

bluebird bio

Forge Biologics

Gain Therapeutics

Ionis Pharmaceuticals

Mirum Pharmaceuticals

Orchard Therapeutics

Vigil Neuroscience, Inc

Viking Therapeutics

Other Resources

Gene Therapy 101

Patient Education – Gene Therapy for CALD and MLD

genehome: Your space to learn about gene therapy

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