ADLD (autosomal dominant leukodystrophy) is a devastating and fatal rare disease that affects only hundreds of people around the world. It causes patients to each day, painfully lose their ability to walk, talk, and even smile.
Rarebase is a public-benefit company that uses cutting-edge science and technology to find off-label uses for FDA-approved drugs that can help patients with rare diseases.
Your donation will go directly towards kicking off a project with Rarebase to find a drug for ADLD. The timeline from now to identifying a drug is 24 months. Can we count on your support?
From the Team
Rarebase has successfully identified drugs for other rare diseases including KIF1A Associated Neurological Disorder (KAND), and is collaborating with over 25 other patient advocacy groups to find drugs for their patients as well. In addition, Rarebase consists of a team of industry experts who have a track record of success across decades of experience in the drug development industry.