Longitudinal MRI imaging in naturally-occurring sheep models of CLN5 and CLN6 disease — two types of Batten disease —…
Articles by Lindsey Shapiro, PhD
Both of Regenxbio‘s gene therapy candidates for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as late…
Female mice with Batten disease caused by a mutation in the Cln8 gene had greater disease severity, including worse behavioral deficits…
A cardiac MRI helped identify heart involvement in a 16-year-old boy with juvenile Batten disease, also known as CLN3…
Activation of a type of channel called TPC2 may restore more normal lysosome function, offering a potential therapeutic approach for…
A targeted epilepsy genetic testing program enabled more children to receive a diagnosis of ceroid lipofuscinosis type 2 disease…
Genetic testing enabled a diagnosis of ceroid lipofuscinosis type 2 disease (CLN2) in six young children with symptoms that…