The ADLD Center raises awareness, supports research, and provides a community for those affected by the rare genetic disease ADLD.
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Autosomal Dominant Leukodystrophy (ADLD) is a rare, genetic disease that causes symptoms similar to multiple sclerosis (MS) e.g., paralysis, speech impairment, bowel dysfunction, etc. ADLD is a fatal disease that slowly progresses - patients often suffer from the disease for several decades after onset.
The mission of the ADLD Center (www.adld.center) is to support research into various aspects of Autosomal Dominant Leukodystrophy. We aim to focus on facilitating research on how the disease works, as well as potential therapies, while helping create a support network to disseminate relevant and helpful information for patients and their families.
Our Work Includes:
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We are currently raising funds for a Natural History Study.
What is a Natural History Study?
A Natural History Study (NHS) is an observational research approach that tracks the progression of a disease over time without therapeutic intervention. These studies are particularly vital for rare diseases, capturing onset, progression, and impact on quality of life. They inform future research, help in the development of treatments, and improve patient care strategies. For rare conditions, where patient numbers are limited and symptoms vary widely, such studies provide critical data that aids in the tailoring of personalized medical approaches and can expedite the regulatory processes for novel therapies.
Why is this study important?
Clinical trials, which are conducted to evaluate the safety, efficacy, and optimal dosing of a treatment in a target population under controlled conditions, usually have placebo groups to differentiate the effects of the drug on those receiving treatment. As ADLD is a rare disease, which makes it ethically challenging to conduct traditional clinical trials with placebo groups. An NHS is able to overcome this ethical hurdle by serving as a real-world control group. It tracks the natural progression of ADLD in affected individuals, collecting invaluable data that can significantly inform and expedite therapeutic interventions.
Benefits of NHS:
How You Can Help:
In order to make this Natural History Study a reality, we need to fund $325,000 over the next two years to cover the costs associated with initiating and sustaining this pivotal study. Here's how you can contribute:
Updates from ADLD Center’s efforts, posted on a regular basis.
