June  2021                                                                                        Volume 32, Issue 6

Register for the 2021 BDSRA Family Conference

BDSRA is looking to add parent and non-parent board seats:

Applications are due August 15th

Note from Board Member Kate Haller about her

journey to the BDSRA Community: 

From my very first family conference in Chicago, I was warmly welcomed by many families.  I was very moved then, and at every conference since then, to hear the stories of families and their children and their amazing resilience.  This is demonstrated so clearly by the number of families who remain involved in BDSRA and return to the annual conference even when their children are no longer with them.

The members of the board bring their own experiences which inform the discussion and decisions at the board.  Just as importantly, members have a variety of skills and knowledge which they all willingly contribute to the board.  This is what I call a working board (not overworked!).  Setting policy and strategic direction is our principal job.  But we also contribute our talents in helping BDSRA achieve its mission every day.

My experience on the board has been very rewarding ,and I encourage others to consider this opportunity as well.

Do you Follow BDSRA on Social Media?

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BDSRA Staff Updates from the BDSRA President 

Morgan DeBoth Promoted to Vice President of Support & Advocacy 

We would like to congratulate our very own Morgan DeBoth for being promoted to Vice President of Support and Advocacy! Morgan has been an innovative force on our team and always ready to accept the newest opportunity. She really felt that in supporting our families enabled her to advocate for them also, so it was a natural fit to re-imagine our BDSRA staff needs. She has done a great job of creating new ways to connect with our families and to help connect our families with each other. We grateful to have her in this role.

Morgan’s new role enabled us to move responsibilities around to better serve our marketing and public relations needs. Communications is the heart of everything we do with our community, so bringing Audrey Albright on board, under Morgan’s direction, as our new Marketing and Public Relations Coordinator is a great way to help us build community. Together they have already increased our awareness, engaged our community and they’ve made great plans for us moving forward.

Morgan is a strong advocate for the Batten Community and a dedicated supporter of our families. Thank you for all that you do for BDSRA, Morgan! 

Community Updates

Thank you for your International Batten Disease

Awareness Day Participation!

 We want to thank you for your participation during our events for International Batten Disease Awareness Day. We appreciate your vulnerability in sharing your stories and pictures to inspire the world. Your dedication to advocacy and support of BDSRA is what gives hope for a cure. Thank you for all that you do! To see featured posts and stories from IBAD 2021, check out the #battenday2021 hashtag on Facebook and Instagram.

Newborn Screening Act Passes the House in Key Legislation

Attention Advocates!

Newborn Screening Saves Lives Reauthorization Act passed the House!

Thank you to Rep. Lucille Roybal-Allard Jaime Herrera Beutler Mike Simpson Congresswoman Katherine Clark for their support of this key legislation!

The Newborn Screening Saves Lives Reauthorization Act will reauthorize critical federal programs that provide assistance to states to improve and expand their newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening.

As an advocate for patients with rare diseases, using your voice can make a difference. 

Resource Corner: Rare Disease Week on Capital Hill

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. While we had hoped to host this event in-person, for the safety of all advocates, Rare Disease Week will be going virtual for 2021. 

Virtual Rare Disease Week on Capitol Hill 2021 will be held July 14th through July 22nd  and will include the same opportunities as in-person Rare Disease Week, plus more!

Registration is live! Visit:

https://everylifefoundation.org/rare-advocates/rare-disease-week/

Upcoming Events for Batten Families 

Research Updates

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.

Research Study Opportunity 

June 2021 Facebook Fundraiser Spotlight:

• Kevin Baptiste
• Sophie Brumbaugh
• Kimberly A Hall
• Matt Koslowski
• Danika Linville
• Morgan Myers
• Melissa Nissen
• Katie Walden 
• Awinna Mae Ward

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! 

Thank You BDSRA Community of Donors!

Your gift makes all the difference in the lives of families.