Beyond Batten

Today is Rare Disease Day. Beyond Batten Disease Foundation is accelerating research, advancing treatment development, and supporting families at every stage of the journey. Learn more at beyondbatten.org.

BBDF is proud to support advocacy group meetings around the world. Sponsoring the Nordic NCL Conference enabled a meaningful gathering of families, clinicians, and researchers from 13 countries, reflecting the international collaboration within the NCL community.

Theranexus is now THX Pharma. This new identity marks an important step forward as the company partners with the Beyond Batten Disease Foundation to advance Batten-1 for regulatory approval and future commercialization. Read the full announcement: buff.ly/87mhHUg

Thank you to the Iron Will Fund and the Feifs family supporters who came together with a goal of raising $100,000 to fund juvenile Batten disease research. Special thanks to the host committee, 1000 and Angela Carlyle who graciously donated her services: angelacarlyle.pic-time.com/tK6TQD6Du7UAx

A case report from the team at Sydney Children's Hospital (SCH) reports on two sisters treated with oral miglustat for 18 months, demonstrating improved visual acuity and clinical stability, with no major side effects. Read the article here: buff.ly/ro0JI7E

#ICYMI — New data shows promising results for Batten-1, with real-world evidence supporting its impact on preserving vision in CLN3 patients. Don’t miss this important update on the progress being made in the fight against Batten disease. Read more: bit.ly/3YNdHXr

Today is Rare Disease Day! 300 million people live with a rare disease, and we’re raising awareness and standing with families impacted by Batten disease and all rare diseases. Visit beyondbatten.org to learn more about how you can make a difference. #RareDiseaseDay

We're delighted to welcome Jonathan Feifs to the Beyond Batten Disease Foundation Board of Directors! With Jonathan’s leadership and insight, we will further our mission of raising awareness and accelerating research for treatments and a cure for Batten Disease.

We need your help! We are collecting info from patients who are currently using off-label miglustat in consultation with their medical practitioner. If your child falls into this category, we would be so grateful for your participation in this survey. buff.ly/3WbjHHg

Phase 2 of the Batten disease research priority-setting process is underway, and your input is requested! This anonymous survey is open to the ENTIRE Batten community and takes 10-15 minutes to complete. The survey deadline is July 17, 2024. buff.ly/3S3xO06

Join us in raising awareness for Batten Disease on Batten Disease Awareness Day! Special thanks and recognition to the families working tirelessly for a treatment or cure for juvenile #Batten disease. Learn more on our website here buff.ly/4aUn8aw.

Share your opinion on Batten disease research needs for all CLN types by completing an anonymous survey. All Batten community members are encouraged to respond. Your feedback will help inform and drive research priorities and investment in Batten. 🔗 ow.ly/Xzml50RKIng

Christiane Benson participated in the Special Olympics this weekend. She brought home three medals and a gold in Western equestrian! ❤️

Fulham for all. 🤍

Thank you @FulhamFC for letting our son William accompany @Bernd_Leno onto the pitch for the big win against Spurs. The @FulhamFCFound does tremendous work for disabled children, allowing them to live their best lives. More on Will's condition here: beyondbatten.org/iron-will/

Today is #RareDiseaseDay! This day aims to raise awareness for people and their families living with a rare disease like Batten Disease. Visit our website at beyondbatten.org to learn more about Batten disease research, the community, and how to get involved.

BBDF is proud to support the CLN3 Natural History Study at the National Institutes of Health (NIH)! View the study here: buff.ly/49k5hJK View a summary of publications arising from this study: buff.ly/3SlKMFN

Check out Christiane Benson, a member of pledge class '20 who has been featured in the Texas Kappa Beta Xi December newsletter! Kudos to Kappa for fostering inclusivity!

What a remarkable day! The 2nd Gaine Invitational Benefiting Wheeler’s Warriors raised $13,500, thanks to the incredible support of friends and family. The funds will assist in crucial research through BBDF, helping kids like Wheeler. Thank you immensely for your support.

Dr. Gary Clark, Chief of Neurology at Texas Children’s Hospital and Lead Investigator of BBDF’s Batten-1 Phase I/II trial presents encouraging preliminary results at the International NCL Congress in Hamburg, Germany today. See press release: beyondbatten.org/press/highly-p…