October 2021 Volume 32, Issue 10
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Advocacy In Action
BDSRA Team attends the NCL 2021 in St. Louis
Pictured (left to right): Dr. Jonathan Cooper, Audrey Albright, Morgan DeBoth, Darlene Royalty, Amy Fenton Parker
On October 6th-10th 2021, BDSRA President & CEO Amy Fenton Parker, Vice President of Support & Advocacy Morgan DeBoth, Chairperson of the Board Darlene Royalty, and Marketing & PR Coordinator Audrey Albright attended NCL 2021 to learn about research updates, listen to future plans for studies and connect with professionals and families in our Batten Community.
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BDSRA President & CEO Amy Fenton Parker leading the NCL 2021 Welcome session with Dr. Jonathan Cooper on night one of the conference.
During her presentation, Amy spoke about the importance of family involvement in research, how to get connected with BDSRA, and the need for a cure.
"The International Conference on Neuronal Ceroid Lipofuscinosis (NCLs or Batten Disease) is the only major international scientific meeting that focuses on advances in scientific, clinical and translational research into these life-limiting inherited pediatric disorders. NCL 2021 will bring together research scientists, clinicians, patient support groups, and the pharmaceutical industry with the goal of the highest standard of scientific and clinical presentations, while still making this science accessible to affected families and other lay persons."
The conference included multiple science presentations, poster sessions, and research update events. Both virtual and in-person attendees were able to ask questions during the Live Q&As following each respective session.
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The main objectives of this conference is to provide a forum to present the latest advances in scientific, clinical and translational research into the NCLs, select presentations on scientific merit from submitted abstracts, rather than inviting established speakers, and promote the highest standard of scientific and clinical presentations, while still making this science accessible to affected families other individuals not directly associated within the scientific field.
NCL typically happens once every two years, alternating between a US location and a Europe location. It is expected the next conference will be held in Eurpoe.
Dr. Cooper and Dr. Mink will be leading a LIVE review session from what was learned at NCL, hosted by BDSRA. Follow our social media accounts to learn more about the TBA time and date of this virtual event. View the official summary completed by Dr. Jonathan Cooper and Dr. Joshua Dearborn below.
For a general overview and summary of NCL, please click the button here:
For an in-depth summary of the NCL Q&A, please click here:
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Morgan DeBoth Featured Panelist at MAGI 2021
BDSRA Vice President of Support & Advocacy Morgan DeBoth was a featured panelist during the WCG MAGI Conference 2021. She joined many other important representatives from the rare disease community.
To view more information about the MAGI conference, click the learn more button:
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Other Featured BDSRA-Attended Conferences in October 2021
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Get Connected with Our Community
BDSRA has a Linktree where you can register for any of our featured events, donate, read the latest news, follow our social media, and more! Check out the new platform by clicking the button.
Click the button to go to our Linktree and get connected:
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BDSRA Launches Annual Appeal
Get ready- BDSRA is kicking off our 2021 Annual Appeal Campaign! Follow our social media for posts about our work and our Batten community. Share our posts with your community.
Invest in the BDSRA mission with a donation to the 2021 Annual Appeal by visiting: https://web.charityengine.net/annualappeal or text "BATTEN" to 243725.
Donate with the link above or click the button:
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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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Upcoming Events for Batten Families
BDSRA staff both develops and attends events important to our Batten Community. Take a look at upcoming November events for rare disease families.
Peer Support Group Leader Training | November 9th @ 6:30p EST | Virtual
Join us Tuesday, November 9th at 6:30pm EST for BDSRA's Peer Support Group Leader Training. This is a training open to anyone currently leading, or who has a desire to lead, a support group for the Batten Disease community. During this time, leaders can discuss best practices, ideas and questions, and learn tools to facilitate your group. We are excited to have you join us and look forward to learning about all the great things currently happening in the community, and how BDSRA can support your efforts!
To register for this event or to send this event to other families, please click here:
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Rare Community Updates
Learn more about this AllStripes x BDSRA collaboration by clicking the button:
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The Impact of Music for Children Diagnosed with Batten Disease
A research study, run by Rebecca Atkinson from Brighton University, UK, is investigating the impact of music for children and young people diagnosed with Batten disease. The research specifically looks into how music therapy and music education programs can support and maintain children’s communication in a school environment.
Anecdotally, parents and staff have emphasised how a child’s language is better when they are singing. Pursuing this further, the study began in 2016, by following children in their music therapy sessions for 3 years, tracking changes in their communication. Findings showed that children’s communication appeared to be maintained when they took part in weekly music therapy or music education sessions (assessment scores plateaued in the communication area over the course of the 3 years).
Follow up research in 2020, created the Music Speaks program which was trialled in the UK with children affected by CLN3. Interviews with staff members talked about how the program was empowering for the children and staff who took part in the trial. Findings demonstrated that from using Music Speaks, children learned how to slow their speech down using beats and draw upon musical phrases to help support their communication. The program also gave staff the skills to support children’s communication through music on a general basis in the classroom.
Ongoing analysis will continue into 2022 with results expected by the end of the year. Research findings will be shared with the BDSRA and the Batten disease community, to help support affected children, their families and educators with musical activities, ideas, and resources.
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A note from Jennifer Siedman from Courageous Parents Network:
CPN has an app! The feature you may find the most helpful is BUNDLE and SHARE tool. This allows you to curate your own library of CPN resources and share it with a person or group. For example, if a member of your community is looking for resources specific to siblings or anticipatory grief, you can use the APP to curate a BUNDLE of videos, podcast and guides to share via text msg or email directly with them.
Another great way parents can use the Bundle and Share tool is in trying to explain the lived family experience to providers, teachers, even friends. For example, if they are having trouble explaining to friends how hard isolating it is to have a child who is seriously ill,they might create a bundle of CPN videos that speak to how they feel and message it to the friend as a way of opening a dialogue about the topic.
Because we know you are all busy, we have some CURATED BUNDLES already in the APP for your exploration and sharing. I think this is a very easy way to personalize your support efforts to members of your community. If you need help sorting the best CPN materials - please reach out to me - I will help you.
Download:
https://apple.co/3AAX2Ht
https://bit.ly/3oVbKa9
Get in on the conversation IN THE ROOM at one of our lived-stream monthly educational events.
Weds., October 27 at 8pm ET - Where Do We Fit In? The Extended Family Experience
There is no perfect protocol for the role extended family members can play. Come hear from a panel of grandparents and aunts who have navigated the complex, emotional and sometimes bumpy landscape of being an extended family member when a child has been diagnosed with a life changing illness.
Weds., November 17 at 8pm ET - Here Come the Holidays
For families of children who had or are living with a serious illness, the holiday season can be a mixed bag. Join members of the Network, to discuss the complex emotions that accompany significant days, strategies for coping, where to find joy and ways rituals may help during the holiday season. After a brief intro this session will be broken up into bereaved and non-bereaved.
Learn more about Courageous Parents Network's current events by clicking the link below:
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A Note from Industry Partners
Taysha Gene Therapies announced their collaboration with University of Texas Southwestern to advance their CLN7 disease gene therapy program. Please read the announcement in the community letter.
Read the full description of the Taysha Community Letter here:
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Breaking News from Neurogene:
"We are pleased to share that the U.S. Food and Drug Administration cleared Neurogene’s Investigational New Drug (IND) application for NGN-101 gene therapy designed to treat CLN5 #Battendisease. Clearing the IND means we have received regulatory approval to move forward in preparing a clinical trial to assess the safety, tolerability, and efficacy of the investigational gene therapy to treat both neurodegeneration and vision loss in CLN5. We are planning to initiate a Phase 1/2 trial in the first half of 2022."
To view this report in full detail on Neurogene's official website, please click here:
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CLN3 Community Update: Theranexus and BBDF move foward with Phase I/II of their clinical trial with BBDF 101.
To view this report in full detail on the Theranexus official website, please click here:
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During the 2nd Quarter 2021 Financial Results and Operational Highlights call on August 9th 2021, RGNX shared the following:
Gene Therapy Using NAV Vectors for Rare Genetic Diseases
RGX-181 for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
REGENXBIO plans to provide a program update by the end of 2021.
RGX-381 for the Treatment of Ocular Manifestations of CLN2 Disease
REGENXBIO plans to provide a program update by the end of 2021.
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October 2021 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 names above. Thank you!
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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.
To have your loved one's name placed in the Illuminator please reach out to Morgan at mdeboth@bdsra.org.
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Thank You BDSRA Community of Donors!
Thank you to the donors last 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
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