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. Work done with Bob Heuckeroth and Alison Snyder-Warwick #battendisease www.biorxiv.org/content/10.1...

Looking forward to the 2025 WORLDSymposium on Lysosomal Diseases 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

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 at Wash U and Bob Heuckeroth at CHOP. Patient led research directly inspired by Batten families. www.science.org/doi/10.1126/...

Happy to migrate over to Blue Sky from the 'other place' please feel free to pass on my info to anyone from the Lysosomal Diseases & Batten Disease research communities