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A small molecule inhibitor of the sortilin protein reduced cellular waste product accumulation and eased neuroinflammation in mouse models of late-infantile and juvenile forms of Batten disease in a recent study. The treatment also completely eliminated tremors in a model of late-infantile…
An experimental gene therapy for late infantile Batten disease showed a good safety profile and appeared to be working as designed in a study done in non-human primates. “The advancement of this potential gene therapy could provide new hope to families with affected children,” Terence R. Flotte, MD, editor-in-chief…
Regenxbio will not move forward with the development of RGX-181 and RGX-381, gene therapy candidates for the treatment of late infantile Batten disease and its ocular (eye) manifestations. Clinical studies were ongoing in Brazil and the U.K. The few patients who have already received a one-time dose of…
Managing fear episodes in people with juvenile Batten disease requires minimizing situations that provoke patients, and increasing their threshold of pain and discomfort, a study reports. Sedative medications and therapies that activate specific areas of the nervous system may also help manage such responses, but solid evidence for them…
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…
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