A Groundbreaking Natural History Study
We are thrilled to announce a collaboration with leading healthcare organizations to kickstart a Natural History Study (NHS) for ADLD. This study is a critical stepping stone to understanding the course of ADLD better and, ultimately, finding a cure.
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
- Inform Future Clinical Trials: With more data on how ADLD naturally progresses, researchers can design more effective clinical trials.
- Patient Stratification: The NHS will help us identify subgroups of patients based on symptom severity, thereby enabling personalized medicine approaches.
- Resource for Global Researchers: The data will be a gold standard worldwide, providing a comprehensive, longitudinal dataset of ADLD progression, which is essential for benchmarking and guiding research efforts globally.
How You Can Help
In order to make this Natural History Study a reality, we need to fund $325,000 over two years to cover the costs associated with initiating and sustaining this pivotal study. Here’s how you can contribute:
- Essential Partner: Your commitment of $500 serves as the foundational support for our mission.
- Impact Partner: A gift of $1,000 enables vital initiatives, amplifying the effectiveness of our work.
- Visionary Partner: An investment of $2,500 catalyzes transformative change, taking our efforts to the next level.
- Leadership Partner: With your generous pledge of $5,000 you’re making a significant commitment to long-term impact.
- Cornerstone Partner: Your contribution of $10,000 will set a benchmark, establishing a legacy of change.
Your generosity could set the cornerstone for a new era in ADLD research. Let’s take this significant step together. Thank you for considering a donation to this transformative initiative.