Thank you for generously supporting our efforts on behalf of the ADLD community!
2022 has been a busy and productive year for the ADLD Center. We want to take this opportunity to share highlights of our recent activities and momentum to support the community. Our key work streams include: Scientific Research, Collaborative Clinical Network, Patient Engagement + Registry, Strategy & Operations, Communications, Fundraising, and Grants.
We hope you will take a moment to read through our recent accomplishments below – this work would not be possible without your investment.
- Funded key research initiative with a gift of $25,000 to support our partner Dr. Quasar Padiath’s crucial ADLD research in the Department of Human Genetics at the School of Public Health at the University of Pittsburgh.
- Officially launched The ADLD Research Hub! We continue our efforts to build a comprehensive ADLD Patient Registry and engage with patients and caregivers to help us understand and serve the needs of the community, in addition to facilitating research.
- Continued creating awareness by leading another well-attended webinar with a number of scientists and patients alike (watch here). There was a very extensive Q&A after the webinar that demonstrated the high level of patient engagement we are seeing.
- Presented at the first ever ADLD scientific conference in the world. Met with leading researchers from various countries including Italy and the Netherlands, and learned about the latest ADLD research. We also informed them about our efforts in order to connect with their ADLD patients and convey our upcoming grant opportunity for ADLD research.
- Attended key scientific meetings including the 2022 Global Leukodystrophy Initiative (GLIA) working meeting, held in Philadelphia, PA, which was designed to have patient advocacy groups collaborate directly with researchers and pharmaceutical industry stakeholders to determine readiness for a clinical trial and path to treatment.
- Investing in research through a partnership with a fellow non-profit org, the Orphan Disease Center at the University of Pennsylvania. We are developing a focused funding opportunity to help meet the needs of our research community. The Request for Applications will launch by the end of the year, and we will award two $50,000 grants to suitable research team(s).
A few more projects that are currently in progress where your funds will have continued impact:
- Exploring a new therapeutic path with n-Lorem Foundation to discover potential treatments for patients with rare genetic diseases. New genetics-based approaches show promise for treating monogenic diseases like ADLD.
- Building out a Research Consortium to hold regularly scheduled meetings with our ADLD research collaborators to address challenges, track progress, and learn from one another on recent advancements. Funding research based on the budget we have available will directly impact the progress we collectively make towards finding a cure for the disease.
- Creating an ADLD Center of Excellence at an academic medical center (AMC) to establish a protocol for tracking disease severity and progression from a clinical perspective. Once we have a proposed treatment intervention, this will enable us to reliably track its efficacy. While AMC’s (i.e physicians, scientists, nurses, care coordinators, etc.) are interested in finding cures for diseases, prioritizing ADLD (i.e. securing physician and team time) over any other rare disease will depend on successful fundraising for ADLD. This makes our fundraising critical to success in treating the disease.
- Serving patients through community events and advancement of the ADLD Research Hub. We must both create a supportive community for patients and caregivers in addition to pushing towards a cure.
We are grateful to have you by our side and look forward to keeping you updated on our progress.
