October's KDA President's Message
Exciting things are happening this month. A pharmaceutical company is about to announce a clinical trial at several U.S. locations, KDA is preparing to conduct a Patient Focused Drug Development meeting that will be used by the FDA in their evaluation of clinical trial results of this and future treatments, and planning is well underway for our annual conference, “KDA 2022,” in San Diego early next month. You can register for the conference by clicking here. We hope to see you there.
The conference will also focus on current SBMA research. Scientists are documenting important findings about the biological processes involved in KD, including new work on the neuromuscular junction (NMJ). The NMJ is a chemical synapse between nerve and muscle cells, and this research is important in the continuing quest to identify the relationship between nerves and muscles in the development of KD. An interesting paper from researchers in Japan documents for the first time the effects of KD on female carriers, and German researchers published results of their study on the relationship between SBMA and cardiac issues. These and other topics will be discussed at KDA 2022, demonstrating the continuing effort to find treatments and a cure for this devastating disease. While science may not always move with the speed we might like, important research into SBMA is continuing to make a difference, and I remain optimistic about the future.
Kennedy's Disease Association
2022 KDA Conference - The Global KDA Family Together Again!
The 2022 KDA Conference will be held on November 9 -11 at the Holiday Inn Bayside in San Diego or virtually on Zoom. While attendance at our annual conferences has greatly increased as a result of the virtual format, we have missed the personal connections and collaboration that occur when we meet in person. This first "hybrid" event will give us the best of both worlds. As always, we are planning presentations on the many facets of Kennedy's Disease: the latest information that contributes to our understanding of it, what works for those who are dealing with it, and what is being done in the research community to develop a treatment. In addition, we will conduct a Patient-Focused Drug Development Meeting in coordination with the Food and Drug Administration so that FDA can hear directly from patients, families, and caregivers about the symptoms that matter most to them and the impact of the disease on their daily lives. For more information about the conference, click here. Preliminary Agenda, click here.
The NIH Launches a New Kennedy's Disease Study
New KD Global Registry
We need your help! We’ve heard from patients and researchers about the need for a patient registry for Kennedy’s Disease also known as Spinal and Bulbar Muscular Atrophy - SBMA. We’re pleased to announce that the KD/SBMA registry is now ready for your data!
The KDA is partnering with the Coordination of Rare Diseases at Sanford Health (Sanford CoRDS) to create a global registry of KD/SBMA patients and carriers. This registry will facilitate KD/SBMA research and will be used to recruit patients for clinical trials. Most important, a robust registry will provide a roadmap for investigators and will help them develop improved treatments and potentially a cure for this debilitating rare disease. Here are a few important things for you to know about the KD/SBMA registry.
- The KD patient registry was developed jointly by NIH and the KDA.
- KD patients and carriers are all urged to enter your data. There is a separate form for carriers.
- Entering data into the registry requires completing the CoRDS profile and two questionnaires, the CoRDS standard questionnaire and the KD/SBMA questionnaire. The entire process should take about 30-45 minutes (less for carriers) and does not have to be done all at one sitting.
- Data can be entered into the registry online, on paper, or by phone with Sanford CoRDS. To learn more, click here.
- There is no cost for entering your data into the registry.
- To protect your privacy, each participant is assigned a Global Unique Identifier that enables de-identification (anonymization) of the data when shared with researchers approved by Sanford Health’s Scientific Advisory Board. Your name and other identifying information will not be shared.
- Providing your consent to give KDA access to your data will allow us to contact you for studies and clinical trials and help improve our understanding about where to drive our research.
- KDA’s goal is to make CoRDS the global repository for KD/SBMA patients. The registry is compliant with U.S. Federal law and the European Union’s General Data Protection Regulation.
Please enroll today, either online or by phone: To enroll online, click here or simply call CoRDS at 1-877-658-9192 and they will enter data on your behalf.
The Kennedy Disease Association Board of Directors
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2022 Rare Diseases and Orphan Products Breakthrough Summit
October 17-18 2022. Washington, DC. This year, NORD is thrilled to welcome the rare family and all community stakeholders back together in-person to Washington, DC for the 2022 Rare Diseases and Orphan Products Breakthrough Summit. Join your fellow rare disease leaders from patient advocacy groups, government, industry, and academia for exclusive access to fresh insights, compelling connections, and expert resources. Don’t miss out on #NORDSummit – registration is OPEN now! For mor information, click here.