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This is a nice way to get the year started off on a good academic pace! Gait analysis of two siblings with atypical CLN2 disease treated with rhTPP1 over 3 years: stable in younger sib nature.com/articles/s4159…

Delighted to see our CLN1 & CLN2 Enteric Nervous System gene therapy paper published in Science Translational Medicine today! Big team effort with many great colleagues Heuckeroth Lab WashU Medicine Pediatrics & directly inspired by Batten families BDSRA Foundation NINDS Tracy VanHoutan science.org/doi/10.1126/sc…

Lysosomal storage disorder mice show bowel transit defects and profound loss of enteric nervous system neurons that can be prevented by gene therapy science.org/doi/10.1126/sc… Science Translational Medicine Jonathan Cooper Heuckeroth Lab

Great to see our ENS gene therapy paper in Science Translational Medicine featured WashU Medicine WashU Medicine Pediatrics WashU Medicine Medical Genetics

Looking forward to WORLDSymposia on Lysosomal Diseases 2025 starting tomorrow. Ewa Ziółkowska (PSDL postdoc) has won a very well-deserved Young Investigator Award and will present her work on dysphagia (swallowing problems) #battendisease

Looking forward to seeing Lysosomal Diseases colleagues and friends WORLDSymposia 2025 starting tomorrow! #battendisease #mps

Pleased to share the latest pre-print from the PSDL highlighting enteric and neuromuscular disease & myopathy in CLN3 mice treated by gene therapy. From talented PSDL postdoc Ewa Ziółkowska Heuckeroth Lab Alison SnyderWarwick ForeBatten Foundation #battendisease biorxiv.org/content/10.110…

Fantastic presentation by PSDL postdoc Ewa Ziółkowska #battendisease ⁦WORLDSymposia⁩ #proudPI

Thank you @bdsra for highlighting our work

Congratulations to all these talented Young Investigators. A special mention for Dr. Ewa Ziółkowska (PSDL). She won this award for a project she devised herself, inspired by Batten families telling her of the swallowing problems their children have BDSRA Foundation family conference

Greatly looking forward WashU Medicine 's Rare Disease Day Symposium tomorrow. Some great speakers covering pre-clinical, translational and clinical research, but most importantly advocacy groups too #RareDiseaseDay (but a week earlier!)

A fantastic way to start Rare Disease day at Otago with the launch of a new theme RARITY and a celebration of RDNZ 25th anniversary. #RDNZ

Today is #RareDiseaseDay2025 This is important for raising awareness of these conditions, but the families we work to help all live the reality of a rare disease every day #battendisease #mps @bdsra BDFA National MPS Society

We are honored and delighted to be among the recipients of the inaugural BDGRI Research Grant round! Very grateful to all including funding partners Anthony Ferrandino drewshope.com Tracy VanHoutan BDSRA Foundation BDFA #BattenResearch

Thanks for featuring our work BDFA BDSRA Foundation Ewa and I are looking forward to starting on this Batten family inspired project! We're even (unintentionally) color coordinated for this video! #BattenResearch

Today its 25 years since the PSDL began! A massive thanks to everyone who has contributed so much to our #Battendisease research over the years, and the Batten families for inspiring it! BDSRA Foundation BDFA

I can confirm that Patricia Dickson is indeed a VERY fine scientist. Her work, insight and expertise speak far louder than any mere qualification! Delighted to work with you Patti! WashU Medicine Pediatrics WashU Medicine Medical Genetics

Great to see Erika here WashU Medicine today & have the chance to discuss all things Batten disease with her this afternoon. There's great value in combining pre-clinical & clinical perspectives to learn from each other to better help affected families #battendisease #NCL

Today is International Batten Disease Awareness Day! This year's T-shirt wisely says: No One Battles Batten Alone! Doing our best to fight the battle for all Batten families, today and EVERY day! #BattenDay2025 BDSRA Foundation BDFA