January 2022
Volume 33, Issue 1
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BDSRA takes on Rare Disease Week on Capitol Hill
Dear Batten Community,
If you aren’t sure where they are, now is the time to find your stripes! I’ve never been one to wear or accessorize with animal print; it’s just not my thing. Then I met our Batten community and discovered this wonderful world of Rare Disease.
I’ve learned that by combining our voices with rare communities like ours amplifies the voice of us all. I’ve learned that an approved treatment serving one community’s disease puts us all in the spotlight. I’ve learned that shared patient and caregiver stories of the journey to diagnosis is affirming for many. I have learned that I am not alone in this rare disease world, and I am so grateful.
Rare Week is Batten’s time to shine on capitol hill. It’s the time to make your voice heard and your face familiar with your representatives. It’s time to wear your stripes like a badge of honor. You can be educated on federal legislative issues, meet other advocates, and share your unique stories with legislators. You can ask us for more information, or you can register below. We would love to have you by our side.
So, I now have a sweater with zebra stripes and my zebra striped Fitbit wristband arrived yesterday. I’m prepared to go to WORLD on behalf of the Batten community, but I’m really ready for Rare Week! How will you join us?
With Hope,
Amy Fenton Parker
President & CEO
Rare Disease Week on Capitol Hill brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. No prior experience is necessary. Registration for this event and all RDLA events are free for all rare disease advocates.
"We were able to learn key information not only for advocating on the hill but how to advocate in our home state. We look forward to continuing the conversation with our local representatives. It was extremely heartwarming to see so many people there, all with the goal of raising awareness of rare diseases."
- Jarrod and Jessica Dicken, 2019
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Do You Follow BDSRA on Social Media?
Want to see more BDSRA content? Don't miss out on upcoming events, information, campaigns and community posts! Follow @BDSRA on Facebook, Instagram, LinkedIn and Twitter to stay in-the-know on all things happening in our Batten Community!
Click below to follow all of our accounts:
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Ask-An-Expert: Research Edition | Feb. 21th, 2022 | 5p EST | Virtual
Learn about clinical updates and ask questions during this LIVE Q&A with Dr. David Pearce. Register or share this event with your community by clicking the link below:
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Quarterly Grief Chat | Feb. 22nd, 2022 | 7p EST | Virtual
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BDSRA understands that each stage of this journey is different, and we are here for you. This is an opportunity for loved ones to gather, share and support one another. Please consider joining us at 7:00pm EST on February 22nd, 2022. Join the event with the zoom link that will be sent via email upon registration. Register or share this event with your community by clicking the link below:
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Stay Golden: St. Patrick's Day Party | March 10, 2022 | 5:30p EST | Virtual
Registration is now open for BDSRA's Stay Golden Saint Patrick's Day Party taking place Thursday, March 10th at 5:30p EST! Join the BDSRA team for a jolly good party with crafts, games, and a festive book! Register by March 1st. to receive a St .Patrick's Party Box! (Boxes for U.S Families only). You can register to receive a box or just attend the virtual party. We would be lucky to see you there! Register or share this event with your community by clicking the link below:
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Please visit our newly added live events calendar:
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BDSRA Welcomes John Patterson to the Team
Welcome to the BDSRA Community, Patterson!
John Patterson is the grandfather of two children affected by CLN3. He has become painfully aware of the impact of the disease on the kids, the SIBS and their parents. He has been attending the Annual Conference since 2016 and knows the value of the interactions and connections which are possible at an in-person conference.
John is a Ph.D. Chemist who lives in Hamilton New Jersey and has become familiar with the science and medicine of Battens, especially CLN3. In addition, he has a strong financial background and is currently serving as Treasurer of his Church.
To see a list of Board members please visit here.
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BDSRA Says Goodbye to Audrey Albright
Wishing you the best of luck!
A note from Audrey to the community.
"I have cherished my time and BDSRA, and have learned so much not only in my position but from team members, families, and industry partners. I want to thank our incredible Batten Families for all of their kindness and support. I love this organization, and will continue to carry the connections I have made close to my heart. I cannot wait to see what BDSRA does in the future for the amazing rare community it serves."
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Advocacy Updates
CLN2 Families: Help us raise the voices of CLN2 children and their families by requesting a Patient-Led Listening Sessions with FDA. BDSRA would like to encourage families who are interested in a session around gene therapy access to apply over the next seven days. By concentrating our effort, we hope FDA will listen to our request and hear our hopes surrounding emerging therapies.
To request a Patient-Led Listening Sessions with FDA visit: https://www.accessdata.fda.gov/.../cder/email/dsp_ESMR.cfm
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HB 927/SB 2900 was introduced into the Mississippi State Assembly last week. This legislation expands newborn screening in Mississippi to ensure that all federal Recommended Uniform Screening Panel (RUSP) conditions are added to the screening panel within three years.
HB 109/SB 242 was introduced into the Maryland State Assembly earlier this month and expands newborn screening in Maryland by ensuring that all federal Recommended Uniform Screening Panel (RUSP) conditions are added to Maryland’s screening panel within two years.
Take action on these initiatives by clicking the button.
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Resource Corner: Tube Feeding
Ensuring that your child is getting proper nutrition while being tube fed is a concern of many in our Batten community. Below are a few resources on tube feeding, we would love to know of any others that parents and caregivers have found valuable. Feel free to email your suggestions to Morgan DeBoth mdeboth@bdsra.org or post them in our Facebook group.
Feeding Tube Awareness Foundation
https://www.feedingtubeawareness.org/
"This website is dedicated to providing pragmatic information for parents and car egivers on handling every aspect of tube feeding and navigating day-to-day life with a child who is tube-fed. Our education materials are written in lay terms by those with personal experience. They are reviewed by medical professionals, and have been distributed by hundreds of children's hospitals, medical practices, and medical supply companies."
The Oley Foundation
"The Oley Foundation is a national, independent, non-profit 501(c)(3) organization that strives to enrich the lives of patients dependent on home intravenous nutrition (parenteral) and tube feeding (enteral) through education, advocacy, and networking. The Foundation also serves as a resource for consumer's families, clinicians and industry representatives, and other interested parties. Programs are directed by the staff and guidance is provided by a board of dedicated professionals and patients."
Feeding Matters
https://www.feedingmatters.org
" Feeding Matters is furthering advances in pediatric feeding disorders by accelerating identification, igniting research, and promoting collaborative care for children and families."
Tubie Friends
"Tubie Friends a non-profit group that is administered by two mothers whose children have feeding tubes and want to use their experience to make life easier for the thousands of children relying on feeding tubes for their nutrition. Tubie Friend Surgeons (TFS) are volunteers who care for or love tube feeders and donate their time and resources. Many of us are still in the trenches with you, which means that sometimes our kids have surgeries, procedures, tests, or hospital stays that takes our time away from Tubie Friends applications and processing."
Tube-Fed Kids Deserve to Eat
"The membership of Tube Fed Kids is primarily parents of past and present tube fed children. The forum is solely for the purpose of sharing experiences and supporting other parents during their own tube feeding/weaning journey. This site is run by parents for parents."
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Research Updates from Industry Partners
BDSRA has some exciting news! We are launching our research locator for families. This tool aims to inform our community about new updates and possible studies available to participate in. Our greatest hope is that this is a simple locator that takes the burden off families who would otherwise be searching through clinical trials.gov. To view our locator, visit: https://www.bdsra.org/research/clinical-studies/
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CLN3 Program update:
Amicus’ Gene therapy team continue their work not only in clinical research but also on finding the best
approach to treat all children living with CLN3.
We will continue to advance our CLN3 Batten disease program. Our current plan is to move forward
with the higher dose, and intra-cisterna magna (ICM) route of delivery pending further Phase 1/2 clinical data from the ongoing study expected in 2022 as well as additional pre-clinical data. These data will inform timeline for start of any pivotal clinical study
CLN6 Program update Amicus will discontinue CLN6 Batten disease gene therapy program following review of long-term extension study data. It was recently determined that any initial stabilization of disease progression at the two-year time point was not maintained through the long-term extension study. Amicus plans to further analyze and share the Phase 1/2 data with the CLN6 Batten disease community and work with the community to support continued research efforts to find better treatments and cures which are so desperately and urgently needed.
If you have any questions, please email: patientadvocacy@amicusrx.com
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No update available at this time.
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No update available at this time.
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No update available at this time.
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No update available at this time.
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No update available at this time.
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No update available at this time.
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Beyond Batten Disease Foundation invites you to read their latest community update here.
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In Loving Memory
Remember with us those we have lost to Batten disease. It's in their honor and memory that we work every day to build a brighter future for families.
Danielle Carbrey
02/16/1993 - 01/24/22
Lydia Headrick
08/2011- 12/2021
Haley Pollman
08/23/2009 - 01/25/2022
To have your loved one's name placed in the Illuminator,
please reach out to Morgan at mdeboth@bdsra.org.
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Sign up for Amazon Smiles
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BDSRA Community Facebook Fundraiser Spotlight
We are so excited each day to log in and see the list of your names growing. Thank you for sharing and supporting our mission on social media! You can visit each fundraiser by clicking on the button above. Thank you!
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Thank You BDSRA Community of Donors!
Thank you to the donors this past month who gave so generously to honor and in memory of loved ones and our community as a whole. We want to acknowledge you for your contributions.
They allow us to keep serving families and advancing the science of a meaningful treatment. Your dollars and support have helped create the leading patient organization in the Batten disease community, and we are dedicated to continue that legacy of children and families.
To view the list of recent donations, please click the button:
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Your gift makes all the difference in the lives of families.
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Batten Disease Support and Research Association
(614) 973-6013 | info@bdsra.org | www.bdsra.org
Batten Disease Support and Research Association
(614) 973-6013 | info@bdsra.org | www.bdsra.org
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