Latus Bio, a biotechnology company developing novel gene therapy candidates for disorders of the central nervous system (CNS), officially launched Thursday, May 1, and announced a lead program for CLN2 Batten disease.

Get all the details by clicking the button below.

The 2024 Annual Family Conference is less than three months away and registration is officially OPEN!

Take advantage of the Early Bird special that includes $5 off the $25 price of each adult registration by June 14.

We listened to your conference feedback last year and the Site Solutions Worldwide team and our conference planning committee sprang into action. Thanks to everyone’s hard work, the FULL AGENDA is now available! Stay tuned for speaker updates and follow our weekly conference posts on our social media channels.

Register, view the agenda, activities for SIBs and Life Goes On, hotel and travel information, and more by clicking the button below.

Our Batten Disease Awareness Fundraising Goal is…$75,000!

We know we can do it! We’ve already raised over 20% of our goal thanks to your support and advocacy.

Register for the virtual 5K, create a fundraising page, join or create a team, order T-shirts, and raise funds leading up to International Batten Disease Awareness Day on June 9th by clicking the button below.

BEHOLD, the 2024 International Batten Disease Awareness Day T-shirt, featuring the winning design by Kennedy Reed from this year’s T-shirt design contest as voted on by the community.

The deadline to order T-shirts is Tuesday, May 7. 

Scroll down to learn more about the story behind this year’s design.

Earn badges as you raise funds for our International Batten Disease Awareness Day fundraiser! Proceeds you raise towards our $75,000 goal will help BDSRA provide compassionate support and advocacy for all types of Batten disease.

Eight-year-old Kennedy Reed’s simple love for drawing and coloring has evolved beyond just the joy it brings her; it has impacted an entire local community through Batten disease awareness thanks to her winning T-shirt design.

Click the button below to read the full story behind this year’s International Batten Disease Awareness Day T-shirt design and the winner!

In his own words, BDSRA Foundation Database Manager Noah Siedman breaks down his takeaways from the RARE Drug Development Symposium, hosted by Global Genes. You can also watch his video summary below. 

What event did you attend and where did it take place? 

I attended the RARE Drug Development Symposium hosted by Global Genes which took place this week in Philadelphia, PA (where I live!).

What new information did you learn, and how can that information help benefit/educate the Batten community?

The first day of the event was dedicated to talking about the role of patients and patient advocacy groups in setting the stage for drug development. That is to say early stages of research and moving into animal models. Most of the sessions were focused on how patient initiatives like Patient Registries and Natural History Studies were the most important things that we can have already in place to help bolster research and speed up drug development. One of the things that was discussed a lot was the idea of clinical endpoints, which is the scientific way of talking about how we measure the effects of a treatment. There are standardized endpoints that organizations like the FDA use, but in the case of many rare diseases, those fall short or aren’t the best metrics. Patient data and input can help researchers and eventually trial designers understand what the right ways to measure effectiveness might be in the context of our patients and community. I also learned that there is a lot of support and resources to help patient-led initiatives like a registry be built and maintained. The idea that the lived experience of our families is something that we can use to help advance the science as effectively as possible is a very exciting one! Furthermore, having all of these resources in place makes developing drugs in our disease space much more appealing for our industry partners. It means that we have already identified the patient population, collected meaningful data, and done the groundwork for designing good clinical trials which will have a greater chance of receiving FDA approval. I also learned about other preclinical work that we can invest in including biobanks, cell lines, animal models, and expanding newborn screening. All of which can help expedite research, drug development, and eventual enrollment of our patients in clinical trials!

Continue reading by scrolling below!

The BDSRA Board of Directors approved February’s meeting minutes during their latest quarterly meeting on Sunday, April 28. View the minutes on our website by clicking the button below.

Whether it’s an urgent need, joy along your Batten disease journey with the help of Fore the Journey, or sending equipment to another family in need, BDSRA is here to support you through our family grants.

The EveryLife Foundation for Rare Diseases offers financial resources and medical resources for the rare disease community.

Learn more by clicking here.

The BDSRA Foundation Family Register is a vital tool that enables us to keep you informed of ongoing Batten disease research, including future clinical research and natural history opportunities.

The information collected in this form is kept STRICTLY CONFIDENTIAL. Your involvement in this survey is entirely voluntary, and you may request to be removed from the list at any time. The form takes just a few minutes to complete and can be accessed by clicking the button below.

What new information did you learn, and how can that information help benefit/educate the Batten community?

The second day was focused more on the clinical trials themselves, and how patient groups can be stakeholders in these trials starting from the very beginning. We learned about different funding models, how both competition and collaboration serve to advance scientific development, and why the patient’s voice needs to be heard early and often. We learned about the value of patient perspective in trial design and how new regulations and technologies can help alleviate the burden on patients who are participating in trials. I also learned about the different steps in the process of drug development, from preclinical research to the regulatory processes before a drug can go to market.

BDSRA Database Manager Noah Siedman spoke at the NewYorkBIO Patient Engagement Summit in March about his experiences engaging with hope as a rare disease sibling. He was introduced by BDSRA Board Treasurer Fred Surrey.

Click the button below to watch Noah’s talk and read his script.

Join us as we celebrate Batten families around the world, advocate, and raise awareness and funds! BDSRA is hosting the 2024 International Batten Disease Awareness Day Virtual 5K on Sunday, June 9th, 2024.

You don’t have to participate in the virtual 5K to advocate for Batten disease. This year, we’re providing a variety of activities for you to raise awareness and funds for Batten disease and BDSRA.

Learn more about the activities, how to donate, and how to get involved by clicking the button below.

The next BDSRA Grief Chat is scheduled for Sunday, June 23, at 7 p.m. EDT.

Prior registration is required for newcomers. If you’ve already completed the Grief Chat form, you will receive an email reminder one week before and the day of. Click the button below for more information.

Where will the path with Batten disease take you? We hope to St. Louis this summer!

Meet us in St. Louis for our Annual Family Conference, which will feature sessions on research, advocacy, caregiver support, grief, and activities for SIBs, bereaved parents, and more! Plus, meet new members of the Batten community and reunite with families you’ve met along your journey.

Click the button below for more information.

Want to see more content from BDSRA? Don’t miss out on upcoming events, information, campaigns, and community posts! Follow @BDSRA on Facebook, Instagram, LinkedIn, X, and YouTube to stay in the know on all things happening in our Batten Community!

Click below to follow all of our accounts: 

Dr. Ineka Whiteman’s April research column will be published on the BDSRA website next week!

Watch our social media channels, website, and your email for publication updates on Dr. Whiteman’s monthly column.

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

Reese M. Donnay | December 11, 2005 – April 9, 2024

Semanur Üngörmüs | December 12, 2004 – April 10, 2024 

Lily Motosicky | September 23, 2007 – April 19, 2024

Mikaylah Sophia Edens | March 19, 2010 – April 28, 2024

To have your loved one’s name placed in The Illuminator and read at the memorial service at the Annual Family Conference in July, please email patrick@bdsrafoundation.org.

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

From copy paper to personnel, $5 Fridays is designed to keep the office moving. The more $5 Friday donations BDSRA Foundation receives for operations, the more money BDSRA can invest in programs, research, and advocacy.

That’s the power of FIVE. 

Designed to give our community control over their stories, fundraising, and donations, our Fam Funds participation is growing!

• Tell your story
• Raise funds for BDSRA
• Choose where donations are directed
• Share with your community