Batten Disease News and Research

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Batten disease (also known as Spielmeyer-Vogt-Sjögren-Batten disease) is a rare, fatal autosomal recessive neurodegenerative disorder that begins in childhood. It is the most common form of a group of disorders called neuronal ceroid lipofuscinosis (or NCLs).
Gene therapy to the inner retina prevents blindness in mouse model of CLN3 Batten disease

Gene therapy to the inner retina prevents blindness in mouse model of CLN3 Batten disease

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Researchers develop new therapeutic approach to treat CLN3 Batten disease

Study unravels CNL6 mystery in Batten disease

Study unravels CNL6 mystery in Batten disease

Research reveals key role of a protein in rare Batten disease

Research reveals key role of a protein in rare Batten disease

Custom-made oligonucleotide therapy for girl with fatal Batten disease

Custom-made oligonucleotide therapy for girl with fatal Batten disease

Researchers develop customized drug treatment to bypass patient’s unique mutation

Researchers develop customized drug treatment to bypass patient’s unique mutation

A Preclinical Solution to Studying Neurological Diseases

A Preclinical Solution to Studying Neurological Diseases

Study reveals connection between two proteins known to be hyperactive in cancer

Study reveals connection between two proteins known to be hyperactive in cancer

OHSU researchers discover disease in monkeys that mimics childhood neurodegenerative disorder

OHSU researchers discover disease in monkeys that mimics childhood neurodegenerative disorder

New therapeutic approach may delay neurodegeneration in rare genetic disease

New therapeutic approach may delay neurodegeneration in rare genetic disease

Raising Awareness of Childhood Dementia

Raising Awareness of Childhood Dementia

FDA approves first drug for treating rare form of Batten disease

FDA approves first drug for treating rare form of Batten disease

Scientists discover treatment that improves symptoms in mouse model of juvenile Batten disease

Scientists discover treatment that improves symptoms in mouse model of juvenile Batten disease

Experts to examine impact of live music on health and morbidity in children with learning disabilities

Experts to examine impact of live music on health and morbidity in children with learning disabilities

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

Gene therapy may be effective method for treating Niemann-Pick disease, type C1

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

Sanford Research scientists develop animal model to study neurodegenerative disease

Sanford Research scientists develop animal model to study neurodegenerative disease

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

NYSCF and Beyond Batten Disease Foundation partner to find cure for juvenile Batten disease

NYSCF and Beyond Batten Disease Foundation partner to find cure for juvenile Batten disease

Expert in gene therapy joins The Children's Hospital of Philadelphia

Expert in gene therapy joins The Children's Hospital of Philadelphia