Batten Disease (CLN5 and CLN7)
Batten disease is a group of rare, inherited diseases of the nervous system also called neuronal ceroid lipofuscinoses, or NCLs. The different subtypes of Batten disease have many common features, but differ in terms of age of onset and rate of progression.
Neurogene is focusing on CLN5 and CLN7, two rare, late infantile (meaning pediatric-onset) and rapidly progressive subtypes of Batten disease. Children with CLN5 or CLN7 typically develop signs and symptoms of the diseases at a young age, including seizures, progressive deterioration in intellectual and motor capabilities, and loss of vision. CLN5 is caused by a variant in the CLN5 gene, which leads to disruption of normal CLN5 protein function. The CLN7 subtype of Batten disease is caused by a variant in the CLN7, also called the MFSD8 gene, which leads to disruption of normal CLN7 protein function.
We are proud to offer LivingBatten.com, a community-focused resource for patients and families. Discover helpful resources and support for CLN5 and CLN7 to guide you along your journey.
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A Natural History Study of Late Infantile Variant CLN5 and CLN7 Disease
Visit ClinicalTrials.gov to learn more about the natural history study that Neurogene is currently conducting for CLN5 and CLN7.