June's KDA President's Message
“June is Bustin’ Out All Over” is a famous song from the musical Carousel. It was one of my favorite songs in junior high school chorus growing up in Michigan. The phrase “Just because it’s June” even today brings back good memories and feelings of optimism. I’m feeling especially optimistic this month for a couple of reasons. First, the NIH Quality of Life survey is underway and going very well. Over 170 people have completed the survey and 45 more people have partially finished it. Thanks to a very generous contribution from a KDA member, the University of Rochester Medical Center will be able to analyze the responses to the survey and develop an SBMA Health Index that can be used to identify outcome measures for use in clinical trials of potential therapies to treat SBMA. The second reason I’m feeling optimistic is that KDA just applied for a grant from the Chan-Zuckerberg Initiative’s “Rare as One” program for rare disease organizations like KDA. RAO grants can be used for any purpose other than research. While we hope to receive a grant, the information we developed to support our application will serve us well in planning future KDA activities regardless of the outcome. Thanks to everyone who participated in the QoL survey and who contributed ideas and suggestions for our grant application. To quote a line from another old song, “This could be the start of something big!”
Kennedy's Disease Association
KDA Awards Grants Totaling $196,200
KDA is pleased to announce 2020 grants totaling $196,200 to the following SBMA researchers listed below. A summary of the projects is provided by clicking here. Congratulations to the recipients!
Characterizing the high prevalence and founder effects for Kennedy’s disease in Indigenous peoples of western Canada
Dr. Gerry Pfeffer (University of Calgary)
Targeting the interaction of poly-Q expanded AR receptor with pVHL to ameliorate SBMA
Antonella Falconieri, PhD (University of Padova)
A drug repurposing strategy to inhibit AR transcriptional coactivators as a therapeutic approach in SBMA
Manuela Basso, PhD (University of Trento)
Unveiling regenerative and metabolic features of SBMA muscle cells to identify new therapeutic targets
Mariarita Galbiatti, PhD (University of Milan)
KDA Annual Conference 2021
October 28th and 29th — SAVE THE DATE! We're going virtual one more time. Evaluations from the 2020 conference were overwhelmingly positive, with many who would not otherwise have been able to attend expressing their appreciation for the opportunity. The virtual format allowed us to reach many more people in a much greater geographical area, and the quality of the talks were outstanding. We hope to do as well or better this year. More details will be coming out soon, but for now, please put Thursday and Friday, the 28th and 29th of October, on your calendar for the 2021 KDA Conference!
Men with KD - Help the NIH Understand
We can’t thank you enough for your responses to the SBMA QoL survey. We have had 172 people that have completed the survey, thank you so much. If you have started the survey and not yet completed, please try to complete the survey as soon as possible. You should get a message, confirming you have completed the survey.
If you have completed the survey, we got your survey with all your answers.
At this time, we are not surveying carriers.
Thank you for your time,
NIH SBMA Clinic
A Quality-of-Life survey is being conducted by the National Institutes of Health (NIH) along with the University of Rochester. They are studying the issues and symptoms that are most important to patients with spinal bulbar muscular atrophy (SBMA). You have the opportunity to provide your insights about SBMA and help researchers ask the right questions. This information will help the NIH and other researchers determine the most important outcome measures to monitor during future SBMA clinical trials.
You are eligible for this research study if you are a man who has Kennedy's Disease and are 18 years of age or older. The survey will take 20-30 minutes to complete and all responses are completely anonymous.
THE GREAT ROAD TRIP - LOS ANGELES TO ROME
KDA and KD-UK are planning an ambitious road trip, in a 29 horsepower 35-year-old Citroen 2CV in 2022, from Los Angeles to Rome from April to July 2022. The car will be shipped to Los Angeles and then shipped across the Atlantic from New York to Southampton. The Citroen 2CV, affectionately known as the tin snail, is a cult car in Europe. It was first manufactured in 1948 and production ceased in 1990. The 2CV has been comprehensively refurbished for the trip. The cost of the 2CV, and its restoration, has already been funded by a generous donor. A seven-day trans alpine cycling event will be run alongside the road trip from Chamonix to Nice in July 2022. Classic Car clubs from across the United States are also expected to join various segments of the American trip. For more information, click here.
Coming Soon - a KD Patient Registry
At the October 2020 KDA-KD/UK conference, several pharmaceutical companies are starting to think about clinical trials of potential treatments for Kennedy’s Disease. Clinical trials are lengthy studies of the effects of a drug or therapy on human participants. They are designed to answer specific research questions.
The good news for SBMA patients is that there ARE MULTIPLE COMPANIES currently designing clinical trials for KD treatment. To prepare for these trials, we need your help fill in the KD patient registry!
The KDA has recently signed an agreement with Sanford CoRDS to build a patient registry for SBMA patients. We ask that you be prepared to enter your personal health history and your experience with Kennedy’s Disease into the CoRDS system that we will use to recruit participants for future clinical trials. Information gathered will be key to patient participation in future clinical trials that, we all hope, will lead to a successful treatment of this debilitating disease.
Thank you from the KDA Board of Directors
COVID19 vaccination in patients with spinal and bulbar muscular atrophy
The Sunnybrook ALS/Neuromuscular clinic at the University of Toronto follows a large number of patients with Kennedy’s Disease (KD). Since patients with KD may experience respiratory impairment and are at increased risk of complication of respiratory infections. The Sunnybrook clinic recommends that KD patients be a priority group to receive vaccination for COVID-19. To read the letter, click here.
Dr. Christopher Grunseich, MD, a staff clinician at the National Institutes of Health has evaluated patients with spinal and bulbar muscular atrophy (SBMA) in multiple clinical studies and mentions in a letter that serious, life-threatening complications due to Covid 19 would be mitigated with COVID19 vaccination. To read the letter, click here.
KDA APRIL NEWSLETTER
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2021 Living Rare, Living Stronger NORD Patient and Family Forum
June 26th and June 27th is the 2021 Living Rare, Living Stronger NORD Patient and Family Forum, a two-day virtual event bringing together rare patients and families to learn, network and participate in wellness and entertainment sessions. With many educational topics offered for undiagnosed, newly diagnosed and long-term patients and caregivers, attendees may customize their schedule to best fit their interests. Your registration also includes a ticket to the Rare Impact Awards on June 28th, an annual celebration honoring the individuals, groups and organizations making a difference by going above and beyond for the rare disease community. For more information, click here.
Rare Diseases and Orphan Products Breakthrough Summit October 18-19, 2021
The 2021 NORD Rare Diseases and Orphan Products Breakthrough Summit will bring the rare disease community together for education, collaboration and networking on October 18 and 19, 2021. For more information on this event, click here.
MDA Access Workshops are a community education program created to provide information and resources on overcoming barriers to a variety of access topics while being specifically built for the neuromuscular disease (NMD) community. These workshops are on-demand. They will allow individuals to navigate at their own pace through online activities, videos, quizzes, and more. The workshops focus on increasing health literacy, empowerment and self-advocacy within the community. For more information or to access the workshops, click here.
Want to help make a difference while you shop in the Amazon app, at no extra cost to you? Simply follow the instructions below to select "The Kennedy's Disease Association" as your charity and activate AmazonSmile in the app. Amazon will donate a portion of your eligible mobile app purchases to us.
How it works:
1. Open the Amazon app on your phone
2. Select the main menu (=) & tap on "AmazonSmile" within Programs & Features
3. Select "The Kennedy's Disease Organization" as your charity
4. Follow the on-screen instructions to activate AmazonSmile in the mobile app