News

Supporters set to rally for Batten Disease Awareness Day on June 9

Fundraising, storytelling, and advocacy are the focus of this year’s International Batten Disease Awareness Day, observed annually on June 9 to bring attention to the group of inherited neurological conditions thought to affect about 1 in every 100,000 individuals globally. The overarching aim of the worldwide event is to…

18-month data on juvenile Batten therapy shows positive efficacy

Treatment with the investigational therapy Batten-1 (miglustat) for 18 months, or about 1.5 years, stabilized motor symptoms in patients with juvenile Batten disease, according to the final results from a Phase 1/2 trial. The progression of motor symptoms was considerably slowed down, as evaluated using the modified Unified Batten Disease…

Webinar to tackle challenges in pediatric rare disease trials

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…