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 ClinicalTrials.gov. ClinicalTrials.gov 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
Patient Education – Gene Therapy for CALD and MLD
genehome: Your space to learn about gene therapy
If you would like to request the addition of your company or other information to this page, please contact us at hope@huntershope.org.