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Recent Posts

• Webinar to tackle challenges in pediatric rare disease trials
• In ‘Blind Momentum,’ Batten disease takes a back seat to bravery
• URMC will use funding boost to bet on Batten gene therapies
• Digital biomarkers may lead to more robust clinical trials for rare diseases
• Regenxbio advancing 2 gene therapies in pipeline for Batten
• Seeking patience in the fight against Batten disease
• Presymptomatic, symptomatic CLN5 sheep aided by gene therapy
• Researchers develop minipig model to study juvenile Batten disease
• Horizon awards grants to 50 rare disease advocacy groups
• Abnormalities in eye tests may help diagnose juvenile Batten: Study