Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 ... Read more
Updates
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Fat Molecule May Help Fight CLN3 Disease, Mouse Study Suggests
Treatment with galactosylceramide (GalCer) — a type of fat molecule — improved motor function, reduced neuronal loss and increased lifespan in a mouse model ... Read more -
Scientists Identify Proteins That May Become Biomarkers of Batten Disease
Scientists have identified a series of proteins that may be useful biomarkers of disease progression for different types of Batten disease. The study, “Analysis ... Read more -
Advancing Clinical Care Recommendations for CLN1 Disease
A marathon is difficult even for world-class marathoners. The best of the best have rough days, days when they turn in a subpar performance, ... Read more -
Complex Fat Molecule Seen to Aid Lifespan, Ease Neuron Loss in Juvenile Batten Animal Study
Treatment with a type of complex fat molecule called galactosylceramide increased lifespan, improved behavioral outcomes and eased neuronal loss in a mouse model of ... Read more
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Single Gene Therapy Injection May Stabilize CLN6 Batten Disease in Children, Early Phase 1/2 Data Show
Amicus Therapeutics‘ investigational gene therapy for CLN6 Batten disease may halt disease progression in ... -
Gene Therapy Prolongs Lifespan, Alleviates Symptoms of Mice With CLN6 Disease, Study Shows
A one-time treatment with an investigational gene therapy prolongs the lifespan in a mouse ... -
Customized Oligonucleotides Treat Seizures in Child with CLN7 Disease in Record Time
Neurologists and researchers have tailor-made an antisense oligonucleotide therapy (ASO) for a 6-year-old girl who ... -
UFC Cheers Young Fan with Batten While Helping to Raise Disease Awareness
The Ultimate Fighting Championship (UFC) lifted the spirits of a boy named Sebastian recently ... -
9 Things We Should Know About the Race to New Treatments
I favor running analogies — even the name of this column pays tribute to ... -
NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, ...
North Carolina’s Indo Jax Surf Charities Helps Kids With Batten Disease, Other Special Needs
News
A small North Carolina nonprofit organization, Indo Jax Surf Charities, has recently gained attention for its set of free programs to help children with Batten disease and other special needs build self-esteem. Since launching these programs, Indo Jax has been featured ... Read more