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).
Advancement of potential gene therapy could offer new hope for children with Batten disease

Advancement of potential gene therapy could offer new hope for children with Batten disease

Study provides insights into the molecular mechanism of Batten disease

Study provides insights into the molecular mechanism of Batten disease

Neural underpinnings of atypical visual sensory behaviors in some children with autism

Neural underpinnings of atypical visual sensory behaviors in some children with autism

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

Oxford-Harrington Rare Disease Centre hosts virtual event in honor of the upcoming Rare Disease Day

Parents become drug developers to save their children’s lives

Parents become drug developers to save their children’s lives

Health care costs for people with rare diseases have been underestimated, study shows

Health care costs for people with rare diseases have been underestimated, study shows

Research shows how mutation linked with CLN3-Batten disease could lead to vision loss

Research shows how mutation linked with CLN3-Batten disease could lead to vision loss

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