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A child with late infantile Batten disease treated with the gene therapy RGX-181 experienced a more than 80% reduction in seizure frequency over the following six months, without any serious side effects. That’s according to data from a single-patient study that were announced by RGX-181’s developer, Regenxbio, in…
This year’s Orphan Drugs and Rare Diseases Conference — the 4th annual such event, taking place Oct. 9-10 in the U.K. — will bring together researchers, academics, healthcare professionals, and other industry stakeholders to discuss the latest findings in the areas of orphan drugs and rare disorders, such as…
A Sept. 28 webinar, hosted by Xtalks, will explore the challenges of clinical research in pediatric rare disorders, along with strategies in decentralizing clinical trials to aid in recruiting and enrolling patients. Registration is required for the free hour-long webinar, titled “The Art of Decentralizing Pediatric…
The University of Rochester Medical Center (URMC) has been awarded a funding boost and will expand its involvement in a national network of academic medical centers dedicated to advancing clinical trials for rare neurological conditions, such as Batten disease. URMC was one of the first medical centers to…
Digital biomarkers are a feasible way to remotely monitor motor function in people with rare neurological diseases, but more work is needed to validate the approach, according to a recent review study. Use of such outcome measures could help make it easier to conduct robust clinical trials for rare diseases,…
A first patient has been dosed in a Phase 1/2 clinical trial testing the gene therapy RGX-381 — one of two in Regenxbio‘s pipeline for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease — as a treatment for the most common form of Batten disease, the…
CLN5-targeted gene therapy safely preserved clinical function, prolonged survival, and prevented brain tissue atrophy in a sheep model of CLN5 disease, a form of late-infantile Batten disease, in a recent study. The benefits were observed in sheep at both pre- and post- symptomatic stages, and the evidence suggests higher…
Researchers have developed a new minipig model of juvenile Batten disease that recapitulates some key features of the human condition, including vision loss and gait abnormalities. Despite certain limitations, scientists believe these miniswine are more translatable to the human disease compared with mouse models of juvenile Batten, also known…
Horizon Therapeutics has awarded grants to help support 50 organizations around the world that are focused on improving the lives of people with rare disorders such as Batten disease. The grants were awarded through the company’s #RAREis Global Advocate Grant initiative, which launched last year with the aim…
Children with juvenile Batten disease frequently show abnormalities in eye tests, such as a bull’s-eye appearance in the macula — the center of the retina, at the back of the eye — and reduced electrical activity in light-sensing eye cells, according to a new study. The findings highlight the…
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