November 2021 Volume 32, Issue 11
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Thank you to Our Giving Tuesday Donors!
Your donation makes a difference.
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Thank you to those that participated in #GivingTuesday on November 30th, 2021! Your donation allows us to support families, fund research, and advocate for legislation important to the rare community.
If you were unable to make a donation for #GivingTuesday, you can still donate to BDSRA's 2021 Annual Appeal. Donate by clicking the button below or text BATTEN to 243725.
Thank you to our #GivingTuesday Participants:
Amanda Major-Bushley
Audrey Albright
Barb Wuebbels
Bonnie Burkhardt
Carson Neifert
Casey Dewey
Chris Hawkins
Courtney Miller
Danielle Yilmaz Dennis
Donna Fogle
Fred Surrey
Gaylene Catalan
James Little
Jennifer Dellomorte
Jessica Padley
Joan Rafferty
Joles Chad
Joseph Frustaglio
Julie Carlson -Noordhoek
Justin Bayer
Kathleen Ruppe
Kelsey Martin
Krista Marie Cartwright
Lana Lee
Lynn Davis
Lynn Spraker
Marcie Minter
Mary Lou Holt
Meghan Dollar
Melissa Munch Becker
Morgan DeBoth
Nancy Foley
Pierre Lariviere
Shane Herman
Shannon Macauley
Staci LaPerriere
Stephanie Burkhardt
Stephen Nowicki
Susan Robles
Tracy VanHoutan
Vanessa Bell
Warren Pfohl
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BDSRA Continues Annual Appeal
BDSRA is continuing the 2021 Annual Appeal Campaign! For the rest of 2021 we will be sharing content and resources important to the Batten community.
Our featured content includes topics such as:
- Support
- Research
- Advocacy
- Staff Thankfulness Week
- Giving Tuesday
- How to Give
- Investors
- What We Do
- Holiday Giving
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 Holiday Party | 21 December 2021 | 6p EST | Free Boxes | Virtual
Join us Tuesday, December 21st, 2021 at 6:00pm EST for the BDSRA Holiday Party! Enjoy fun festivities and holiday crafts with the BDSRA team!
Choose a "Holiday Box" ticket to receive a free box of holiday fun during the party! This box will include crafts, games, and a special holiday book to read together! Register by December 8th to be able to receive the Holiday Box. One box is needed per participant, U.S. families only.
We hope to see you at the party!
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AAE: Research Edition | Q&A | 20 December 2021 | 5:30p EST | Virtual
Join us for Ask-An-Expert: Research Edition on Monday, December 20th at 5:30pm EST with Dr. David Pearce. Ask questions and learn about research updates important to our Batten Community. Register with the link and join from Facebook Live or the webinar link that will be sent via email upon registration. Register with the button below.
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Grief & the Holidays | 16 December 2021 | 7p EST | Virtual
For families of children who had or are living with Batten Disease, the holiday season can be a difficult time. Join members of the BDSRA community Thursday, December 16th at 7:00p EST to discuss the complex emotions that accompany significant days, strategies for coping, where to find joy and how to honor your family traditions. Register today and look for a confirmation email with the event link to join the day of the event
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BDSRA Community - We Want Your Feedback!
2022 Conference Content Survey
We are so excited to see everyone for the 2022 BDSRA Family Conference in Cleveland, Ohio! We plan on having a limited hybrid option so people unable to attend in person can still be part of the conference. Please fill out the two-question form below to let BDSRA know how you are attending and what content you would like to see at the conference. Thank you!
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Community Needs Assessment
Our 2021 Community Needs Assessment is live. This assessment is used to inform BDSRA and guide us in all we do in our Batten community. This 20- minute survey gives us insight into our community so that we can better plan and be proactive around community needs. We thank you for your time and willingness to share. Click the button below to take our survey. Thank you!
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BDSRA Community Updates
Thank You & Best of Luck to Colin Monteith!
BDSRA would like to thank Colin Monteith for all of the great work he completed during his time at BDSRA as he continues on to new endeavors in his career path. We greatly appreciate the three years Colin dedicated to the Batten Community and wish him the best of luck in the future!
"Whether it was “bad Dad” jokes or witty puns, Colin always kept us guessing in the office. He knew our donors well and had a true heart for the mission of BDSRA. Colin’s wealth of knowledge and his diligence has set BDSRA up for our continued success, and I am grateful for his commitment to see this year through." -BDSRA President & CEO Amy Fenton Parker
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BDSRA Says Goodbye & Thank You to Two Board Members
BDSRA thanks Brock Benroth and Bob Jensen for their dedication and commitment to the Batten community during their time on the board.
"Being on the board was a different way that I could help the BDSRA and honor Kate's legacy. It was very interesting to get to witness some of the inner workings firsthand, and to help make the important decisions that affect some many families and affected children."
- Brock Benroth
"I regret to have to leave you because my friendship has deepened with you but that will stay and my passion to continue to find the cure. So as Frank Sinatra said, 'regrets I have a few' - but hope BDSRA and the Board has felt my presence and contributions."
-Bob Jensen
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BDSRA President & CEO Press Release
Click the button to see the official press release from BDSRA President & CEO Amy Fenton Parker for November 2021.
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Advocacy Updates
Everylife Foundation
"ODTC Update - Last week the House passed the Build Back Better Act (HR 5376). Unfortunately, the final bill passed by the House contained the provision (Section 138141) that would limit the Orphan Drug Tax Credit (ODTC) to "the first use or indication with respect to which a drug for a rare disease or condition is designated under section 526 of the Federal Food, Drug, and Cosmetic Act."
We continue to be concerned that limiting the credit – already reduced from 50 to 25 percent – for qualified clinical testing would diminish interest in evaluating a drug that has been approved for both rare and non-rare conditions for use to treat another disease or condition.
Cures 2.0, H.R 6000 - This bipartisan legislation was recently introduced in the House by Representative DeGette (D-CO) and Representative Upton (R-MI). This bill reflects the urgency and opportunity of the rare disease community to build on the momentum and successes of the 21st Century Cures Act (2016). The policies in Cures 2.0 will continue to improve lives and health outcomes by shortening the diagnostic odyssey, expanding scientific and data innovation, enhancing regulatory infrastructure, ensuring that patient experience informs decision making, and creating pathways to equitable access to novel treatments."
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BDSRA's Call to Action: CLN2 Community
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CLN2 Batten community, we are asking you to engage FDA to request an opportunity in which families could speak directly to the FDA about unmet medical needs, the push for new therapies, etc. This would allow for your current experience to be heard. BDSRA has requested a session and we encourage you to raise your voice as well over the next 7 days.
Click the button to learn more about patient listening sessions.
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Research Updates from Industry Partners
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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"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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Programs for the Treatment of Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
- REGENXBIO is continuing to evaluate the path forward for RGX-181 for the treatment of CLN2 disease and plans to provide a program update in 2022.
- REGENXBIO is conducting additional preclinical studies of RGX-381 for the treatment of ocular manifestations of CLN2 disease and is in discussions with regulatory agencies. REGENXBIO plans to provide a program update in 2022.
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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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No update available at this time.
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November 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.
Teague Joseph Niebrugge
October 10, 2005 - November 2, 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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