September 2021 Volume 32, Issue 9
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Advocacy In Action
BDSRA Team attends the 50th CNS Annual Meeting
The 50th Golden Anniversary Meeting of the Child Neurology Society was held September 29-October 2, 2021 in Boston, MA. The meeting featured the usual high level educational content with the theme “Child Neurology: Past, Present and Future.” Batten Disease Support and Research Association President & CEO Amy Fenton Parker and Vice President of Support & Advocacy Morgan DeBoth consider representing Batten Disease at the conference as an honor.
Amy and Morgan have met friends and allies in research, clinical and industry, as well as introducing other attendees to the Batten community. In many sessions presenters have included Batten as an example in disease, therapy, and history. The excitement for the future of treatment is projected by the presenters, and the awareness and advocacy for rare patients is also prevalent. “To be present in person to learn about where neurology began and where the science is going is a gift of our donors that we can share with the wider community,” said Amy Fenton Parker. Learn more about CNS 2021 by clicking the button below.
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BDSRA Offers Resources for Family Grants
BDSRA has multiple grants and services available to assist Batten Families including the Batten Family Help Grant, BDSRA Equipment Exchange, and Fore the Journey Fund- a collaboration with ForeBatten Foundation. Please click the button below to view and apply for the featured grants available. For any questions on grants and resources please email BDSRA Vice President of Support & Advocacy, Morgan DeBoth, at mdeboth@bdsra.org.
For more information on resources available or to apply, please click here:
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Get Connected with Our Community
BDSRA has a new 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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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 October events for rare disease families.
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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 Monday, October 4th 2021. Register to attend by clicking the button below. Join the event with the zoom link that will be sent via email upon registration.
To register for this event or to send this event to other families, please click here:
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Join us for Ask-An-Expert: Lab Edition on Tuesday, October 19th, 2021 at 5:00pm EST with Brandon Whipple, Director Surgical Research Center and Director Animal Research Center at Sanford Research. Lab Edition includes an informational session on how labs function and a LIVE Q&A to follow. Register by clicking the button below.
To register for this event or to send this event to other families, please click here:
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Join us Thursday, October 28th 2021 at 5:30pm EST for the BDSRA Boo Bash! Enjoy a spook-tacular party of family fun with the BDSRA team!
Choose a "Boo Bash Box" ticket to receive a free box of boo-tastic supplies to use during the party! This box will include crafts, games, and a ghostly-fun book to read together! Register by October 15th to be able to receive the Boo Bash Box. One box is needed per participant, U.S. families only. Register with the button below.
We look forward to seeing you at the event!
To register for this event or to send this event to other families, please click here:
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Looking to attend NCL 2021? BDSRA has partnered with a generous donor to allow assistance for U.S. families wanting to attend the International Conference on Neuronal Ceroid Lipofuscinosis. This conference will bring together research scientists, clinicians, research support groups, pharmaceutical industry partners and Batten Families. For more information on how to apply for assistance, please email mdeboth@bdsra.org.
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The International Congress on Neuronal Ceroid Lipofuscinosis event will take place at Washington University, St Louis, October 6-10, 2021 and will be a 'hybrid' conference. This means both in-person and online-only registration options will be available. There is also a reduced registration fee for families available!
This a parent-friendly conference; decoding the science for everyone to understand. BDSRA's Amy Fenton Parker and Morgan DeBoth will be in attendance. All are welcome!
More details can be found by visiting: www.ncl2021.org
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NORD Rare Summit 2021: Virtual | October 18-19, 2021
Each year, the NORD Summit, one of the largest multi-stakeholder events in rare disease, brings together participants spanning the rare community, including rare disease experts and leaders from patient advocacy groups, government, industry, and academia to discuss the most current and critical topics related to rare diseases and orphan products. Registration is now open!
To learn more information or to register, click the button:
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BDSRA Board Spotlight
A note from Darlene Royalty, Chairperson of the BDSRA Board of Directors:
My husband, Randy and I are from Ursa, IL. We had three girls with CLN3. Amber died at age 28, Sandy died at age 21, and Sarah died at age 24, so we watched them grow and blossom. It was my sister, Connie Vermeire, a registered nurse, who researched all about Batten Disease when the doctor told us we were going to lose our girls.
It was the summer of 1996 when Randy and I went to our first conference. It was the worst time of our lives and yet it was what saved our lives. You never forget the person who picks you up and shows you the way. Nancy Peterson, from California was that person. After talking to her while waiting for our ride to the airport we found out we were not alone and were not so different. We have attended twenty conference over the years. To us it is like coming home to family.
I am reminded of all the hugs and tears that have been shared by all the different people we have met over the years. If you have not experienced the fear and terrifying feelings, you can’t understand it. But our Batten family knows what we were going through.
I have always wanted to be on the board. I have strong ideas about the support part of the organization. I want to help others to take care of the caregiver. I know that research is important, but not everyone will have the opportunity to have a treatment. Our families need the day-to-day support. I try to live that concept every day. I chose to try to live through this and to make it to the other side, after the girls were gone. But I did not have the time when the girls were here, and I knew it would cost us financially. The position on the board is a volunteer job. You have to pay your own expenses to the meetings and hopefully you will support the organization monthly.
I was at a winter board meeting, and we were talking about starting the family help fund, Donna Fogle suggested that if each board member sent $25 to $30 on a monthly basis, we could fund this program. I have my donation taken out of my bank account monthly and I don’t have to worry about it. Donation amounts are between you, your family and God. But my folks always made a point to tithe at church and so I do my tithing with Batten Disease. Randy and I have also set up a donation in our will. We have no other children, so this is our way of making a difference.
Families dealing with Batten Disease are on their own journey. No two are the same. There are people out there that would make a terrific addition to the Board of Directors. We are always looking for someone to fill a position with special expertise to move Batten Disease forward here in the United States and to make us a leader worldwide. Everyone currently on the board has improved our organization and we are stronger for it. I am proud to be the Chairperson of the Board of Directors. Our board works well with our President & CEO Amy Fenton Parker and her great staff. I can’t wait until the 2022 Conference in Cleveland where we can once again meet in person. If you need anything, please call our office. I hope to see you all in Cleveland.
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During the summer quarterly BDSRA Board Meeting, Board members, BDSRA leadership, and a few batten families gathered to tour Sanford health in Sioux Falls, SD. The board is excited for continue its work and support of the BDSRA mission. The board looks forward to reviewing applications and goal setting as they look toward 2022.
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How to Host a BDSRA Fundraiser
Thinking about hosting a fundraiser for BDSRA? We would love to support your fundraising event and help with preparation. If you have questions or are considering hosting a fundraiser of your own, please email mdeboth@bdsra.org. See the Batten Community Spotlight below to view some of the recent fundraisers for BDSRA held by our amazing community members!
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Batten Community Spotlight
Thank you to community member Bekah Murphy for creating a new annual BDSRA fundraiser: Bike For Batten! See below for details and results on how the bike event went. Keep an eye out for the #BikeForBatten event next year.
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"These rides are inspired by my two courageous and amazing nephews. They are Titus and Ely and they both have Batten disease. Titus ran into the arms of Jesus at the age of 6. His parents, Danny and Bekah and little brother, Ely, miss him tremendously. Just 2 weeks after Titus passed, Ely had the life giving opportunity to begin an enzyme replacement treatment. This incredible treatment was created by a team of experts who desire to help Batten children have a prolonged and high quality of life. Ely is living proof that the research being done is indeed working! He is still able to walk, eat, and communicate.
This would not be possible without this enzyme replacement therapy. We are forever grateful for the work being done. The research teams and doctors are currently in the midst of finding a treatment to prevent blindness in children as well as gene therapy. There is a lot more work to be done and it is exciting! All funds raised during Bike for Batten will go directly to BDSRA (Batten Disease Support and Research Association). You WILL make a difference in the lives of these amazing children."
-Bekah Murphy, Bike For Batten
If you would like to donate to BDSRA on behalf of Bekah's fundraiser event, please click the button.
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Thank you to community member Allen Severance for creating a collaborative fundraiser between BDSRA and Rise Up For Grant! Rise Up for Grant held a Bass Fishing Tournament and a #SwimbaitForBatten Raffle, complete with a taco truck and prizes. Thank you to Rise Up for Grant, Allen, and his family for their support!
If you would like to donate to BDSRA on behalf of Allen's fundraiser event, please click the button.
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Thank you to the Beedle family for inviting BDSRA to their fundraising event! We were there to advocate, but were gifted proceeds from their 5k and lemonade stand! Pictured: BDSRA VP of Support & Advocacy Morgan DeBoth with friends and family of the Beedle Family.
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Thank you to community member and BDSRA board member Fred Surrey and his family for putting on their annual yard sale to support BDSRA! This was a multi-day event they held in their hometown. BDSRA would like to personally thank the Surrey family for their years of support and dedication through their annual fundraiser, and all that they do for the community.
If you would like to donate to BDSRA on behalf of the Surrey's fundraiser event, please click the button.
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A Note from Industry Partners
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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Natural History Study enrolling in person.
If you have concerns about traveling, please reach out to Morgan DeBoth at mdeboth@bdsra.org to coordinate alternative participation opportunities.
https://clinicaltrials.gov/ct2/show/NCT03822650?cond=cln5&draw=2&rank=1
Caregiver Focus Groups for CLN5
Dates will be determined by the families’ availability. View article here.
Neurogene announces the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to CLN5 Batten Disease Therapy. This announcement follows the FDA’s Orphan Drug Designation which demonstrates continued progress in establishing a Batten disease franchise.
To read the full press release click here.
Neurogene is currently working with RTI Health Solutions (RTI‑HS), an independent, nonprofit research organization to better understand the disease progression of Batten disease subtype CLN5 from the caregiver’s perspective.
Caregivers of children (either currently living or deceased within the past 12 months) with Batten disease subtype CLN5 who are eligible for this study (Qualitative Interviews With Caregivers of Children With CLN5-Specific Batten Disease) and live in the United States will be invited to participate in a telephone interview with two RTI‑HS interviewers lasting approximately 90 minutes. Interview participants will receive an electronic gift card (e.g., Visa, Amazon, or other online store of choice) for $150 in appreciation for their time.
During the interviews with RTI‑HS, caregivers will be asked to describe their child’s and their own experiences surrounding the journey to and following diagnosis, including observed signs and symptoms of the disease, delays in any of the developmental milestones, and perceived impacts of Batten disease subtype CLN5 on the child, caregiver, and family.
The discussion will be audio recorded and transcribed to ensure the interviewers capture the important points that participants share and to help write a report summarizing the results of the interviews. Participants’ names and any other identifying information will not be associated with the transcripts. The audio recordings will be destroyed once a final report is written.
If you are interested in participating in a telephone interview, please call Danielle at L&E Research at 720-647-3228 who will ask you a few questions to confirm that you qualify for this study. If you qualify, L&E Research will schedule you for an interview at a day and time that is convenient for you.
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September 2021 Facebook Fundraiser Spotlight:
- Katie Allbee
- Stacey Ardizzone
- Tara Kiley
- Amy Lauren
- Jessica Marie
- John McAuley
- Martha Pankratz
- Nicole Pike
- Martha Shipley Pankratz
- Elizabeth Vaughn
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.
Emirican Abay (8.2021)
Justin Moore (12.2.1990 - 8.26.2021)
Jagger Null (11.16.2010 - 8.30.2021)
Michael Rochester (1998 - 8.28.2021)
Austen Weaver (9.24.2001 - 8.26.2021)
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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