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).
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

Study addresses how location and sex can affect mouse models in scientific research

Study addresses how location and sex can affect mouse models in scientific research

Enzyme therapy slows progression of Batten disease in dogs

Enzyme therapy slows progression of Batten disease in dogs

Combination therapy may improve life span and motor function of infants with Batten disease

Combination therapy may improve life span and motor function of infants with Batten disease

Study reveals what causes lysosome to overflow in Batten disease

Study reveals what causes lysosome to overflow in Batten disease

Rochester receives $1M FDA, BDRSA grant to begin first controlled clinical trial in Juvenile Batten disease

Rochester receives $1M FDA, BDRSA grant to begin first controlled clinical trial in Juvenile Batten disease

Genetic mutation linked to Kufs disease

Genetic mutation linked to Kufs disease

Scientists use new technologies to identify genetic cause of Kufs disease

Scientists use new technologies to identify genetic cause of Kufs disease

Scientists detect several biomarkers to screen active drugs for Batten disease

Scientists detect several biomarkers to screen active drugs for Batten disease

Raptor reopens enrollment in DR Cysteamine Phase 3 trial to treat patients with nephropathic cystinosis

Raptor reopens enrollment in DR Cysteamine Phase 3 trial to treat patients with nephropathic cystinosis

Raptor completes enrollment in DR Cysteamine Phase 3 clinical trial in cystinosis

Raptor completes enrollment in DR Cysteamine Phase 3 clinical trial in cystinosis

Comprehensive guide to current practice of intraoperative neurophysiology

Comprehensive guide to current practice of intraoperative neurophysiology

OHSU to lead next phase of HuCNS-SC trial for NCL in children

OHSU to lead next phase of HuCNS-SC trial for NCL in children

StemCells initiates HuCNS-SC second clinical trial for Batten disease in children

StemCells initiates HuCNS-SC second clinical trial for Batten disease in children

StemCells provides update on PMD Phase I clinical trial

StemCells provides update on PMD Phase I clinical trial

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

StemCells second-quarter net loss decreases to $4.61 million

StemCells second-quarter net loss decreases to $4.61 million

NIH grants $14.85 million to complete two floors of NRI at Texas Children's Hospital

NIH grants $14.85 million to complete two floors of NRI at Texas Children's Hospital