March 2023                                                                            Volume 34, Issue 3

Courage. I have learned what courage truly is from you, our Batten Community. It is waking up every day knowing a battle lies before you but choosing to show up anyway. Our Batten community members show up for their families and support one another. You courageously give your time, talent, and treasure to BDSRA. Thank you for all you do and for your inspiring example.

The BDSRA Foundation team continues to accomplish much, often in the face of unique challenges, but we’re so fortunate to have your support. Our board, staff, and community members have filled the gap created by the absence of a Support & Advocacy person. So many have provided virtual programming, and many are helping us plan the 2023 Annual Family Conference in Denton, Texas, July 14-16. Our important sponsorship outreach continues…who do you know that might sponsor the conference? Feel free to email suggestions to Sponsorship@BDSRAFoundation.org.  

Preparations are underway for International Batten Disease Awareness Day, June 9th. Watch our social media for details, rules, and a template to participate in our T-shirt design contest. We’re helping teams with engagement ideas and events in their own communities. What special events do you have planned? Please like and share our June 9th posts!

Below are but a few more examples of ongoing projects you will soon hear more about:

• resurrecting a 2019 research grant
• revitalizing the Batten Centers of Excellence
• hiring a new Science Officer for BDSRA
• identifying the next conference sight

Some accomplishments we’re celebrating:

• On January 21 in Chicago, we welcomed four new board members
• We recently packed up and moved our office to a smaller space. This will save the BDSRA Foundation more than $27,000 this year
• We planned and hosted a virtual Valentine’s Party for more than 40 families

• We have processed 17 grants in these three short months

Recently, Patrick stepped up to represent Batten Disease at Rare Disease week on behalf of BDSRA for me, as I was facing a personal challenge.

I was recently diagnosed with Non-Hodgkin Lymphoma. My health challenges have and will continue for several months. This will stretch our capacity and require the help of the board and volunteers. Our team has been doing a fantastic job of working together during this difficult time, and I am so grateful. My prognosis is very good, and my second treatment is already here! I have four more treatments to go, with the last just two weeks before our conference.

I share this because I want you to know that, thanks to our Batten Community, I and your foundation team have the courage to march forward, to ask for help when needed, and to apologize when we’re not immediately responsive. We remain determined to serve families affected by all CLN types, who rely on our compassion and ability to connect them with the resources they need, and with each other.

The BDSRA Foundation is deeply invested in the cause of BDSRA. I ask that you continue your courageous support of our operations. Now is the time to invest in our foundation, so that together, we may continue the good work that we have planned.

Amy Fenton Parker

The fight for gene therapy has been a long, uphill battle for the Batten community that has featured many losses and few victories.

It’s a battle that Suzette James is eager to help turn around. James, a BDSRA Board Member and Batten mother to Maya and Xavier, who are both affected by late-onset CLN2, will have another opportunity to help do just that.

She’ll be a featured panelist in the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) public virtual workshop on Thursday, April 13, from 11 a.m. to 3 p.m. ET. The workshop is titled, “Clinical Trials: The Patient Experience.”

Click the button below to read the full story and register to attend the event!

Our 2023 Rare Disease Week recap is live!

Visit our website, go to the Advocacy tab, and click on Rare Disease Week in the drop-down menu or click the button below to read and watch summaries from Rare Disease Day and Rare Disease Week.

The summary includes video summaries of Rare Disease Day events from BDSRA staff and a board member as well as Patrick Kotnik’s (BDSRA Marketing & PR Coordinator) meetings with staffers representing Ohio legislators.

From a storybook ending at Rare Disease Day at NIH to a road for optimism, Kotnik details his takeaways in writing from this year’s Rare Disease Week on Capitol Hill. This piece is also included in the recap and was published in the EveryLife Foundation’s monthly newsletter, The EveryLife Rare Report.

We want to say a special thank you to Donna Fogle, BDSRA Board Secretary and bereaved Batten mother to Kourtney.

Donna attended Rare Disease Week on Capitol Hill and met with numerous legislators and staffers to help gather support for RDLA legislative initiatives, stress the importance of reauthorizing newborn screening, and begin the process of asking for a federal proclamation to declare June 9th as International Batten Disease Awareness Day annually.

We are grateful for Donna and her continued advocacy efforts for the Batten community!

Our Database Manager Noah Siedman traveled to Columbus from Massachusetts to meet the staff in person, see the new office, and continue working on various projects and day-to-day tasks.

We’re grateful for Noah and can’t wait to see him again this summer! Thank you to Drew’s Hope for making this visit possible.

The 2023 BDSRA Annual Hybrid Family Conference will take place from July 14-16 at the Embassy Suites by Hilton Denton Convention Center in Denton, Texas! Book a room by clicking the button below!

Follow our website and social media channels for both in-person and remote registration information this spring.

If you live in Texas, be sure to advocate for the state’s RUSP Alignment bills!

Urge your state legislators to support H.B. 2478 and S.B. 1697. This legislation will eliminate delays in screening for diseases, ensuring that babies born with life-threatening diseases are diagnosed and treated at the earliest age possible.

Click the button below to visit the EveryLife Foundation website and take action!

Rare Disease Week has passed, but the advocacy doesn’t stop!

Want to get involved in advocacy? Here are some advocacy tips from Batten mothers.

Tracy VanHoutan, a bereaved Batten father to Noah and Laine and Batten advocate, highlights different ways to advocate and the crucial role advocacy plays in your Batten journey.

Sharon King, a bereaved Batten mother, Batten advocate, and President of Taylor’s Tale, spoke during a panel at Rare Disease Day at NIH.

“Tell those stories — it’s the most important thing you can do, and everything else will grow from there.”

Click the button below to watch Taylor’s story by the Foundation for the National Institutes of Health.

Last month, RareDiseaseDay.org featured a Batten family’s story.

Get to know the Palermo family and Jennifer and Philip’s daughter, Amelia, as Jennifer details their journey with CLN1 Batten disease.

The BDSRA Foundation is committed to supporting and advocating for those affected by Batten disease to raise awareness and give this community a platform to share their stories.

Sharing your story allows BDSRA to help raise awareness for Batten disease and advocate for improvements and developments for our three pillars — Support, Research, and Advocacy.

Please contact BDSRA Foundation Marketing & PR Coordinator Patrick Kotnik at patrick@bdsrafoundation.org if you’re interested in sharing your story with BDSRA.

Shared your story with local media? Let us know! Email Patrick and we will share!

In case you haven’t heard, BDSRA Foundation has moved offices!

With that comes a new address to send mail to BDSRA. For any mail and shipments to BDSRA, please use the address below. 

P.O. Box 30049
Columbus, OH 43230

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, Twitter, and YouTube to stay in the know on all things happening in our Batten Community!

Click below to follow all of our accounts: 

Courageous Parents Network, a non-profit organization and educational platform that orients, empowers, and accompanies families and providers caring for children with serious illnesses, will be hosting another virtual workshop on Wednesday, April 12th titled. “Understanding and Addressing Medical Trauma.”

This workshop is free to attend and will take place on Zoom.

Join us at FDA CBER’s annual patient engagement workshop on Thursday, April 13 at 11 a.m. ET.

Hear inspiring stories from patients, caregivers, and advocates, including BDSRA Board Member and Batten mother Suzette James, who have navigated gene therapy clinical trials, and learn about the risks and benefits of these studies.

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a virtual public listening meeting to solicit input on methods and approaches for capturing post approval safety and efficacy data for cell and gene therapy products.

Attention Medicaid and CHIP consumers, renewals are coming back! Many were paused due to COVID-19.

Be sure you get your renewal letter by making sure your state has your current mailing address NOW.

Research Updates from Industry Partners

Visit the Research tab on our website to keep up with new and investigational studies! bdsrafoundation.org/research/clinical-studies/ 

No update available at this time.

No update available at this time.

No update available at this time.

No update available at this time.

No update available at this time.

No update available at this time.

No update available at this time.

No update available at this time.

March 2023 Facebook Fundraiser Spotlight:

Thank you for sharing and supporting our mission on social media! You can visit each fundraiser by clicking on the names above. Thank you!

In Loving Memory 

Remember those we have lost to Batten disease. We work daily to build a brighter future for families in their honor and memory.

Johannes Nienhaus | May 18, 2016 – March 10, 2023

Max Reid | September 14, 2007 – March 12, 2023

Ella Dawn Nakvinda | March 8, 2019 – March 27, 2023

To have your loved one’s name placed in The Illuminator please email info@bdsrafoundation.org.

Thank You BDSRA Community of Donors!

Thank you for investing in our mission. We exist to provide support, research, and advocacy for families with all forms of Batten Disease. We want to acknowledge the donors who gave so generously last month to our work. Together, we are Batten Advocates for a Cure.

To view the list of recent donors, please click the button below!

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

BDSRA Foundation

P.O. Box 30049
Columbus, OH 43230

(800) 448-4570 | info@bdsrafoundation.org | www.bdsrafoundation.org

View Message Online Link