Margarida Maia, PhD,  science writer—

People with juvenile Batten disease treated with the small molecule Batten-1 (miglustat) for 18 months in a clinical trial, and who continued with treatment outside the study, showed no signs of deteriorating vision, a common disease symptom, the company developing Batten-1, Theranexus, announced. All patients completed…

With its RareCare Patient Assistance Program, the National Organization for Rare Disorders (NORD) helps to cover healthcare costs that aren’t paid by health insurance — and the program is available to people with Batten disease in the U.S. who may be struggling to pay bills directly related to…

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…

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…

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…

The Batten Disease Support and Research Association (BDSRA) is challenging people to walk, run, or bike a virtual 5K anytime from now through June 9 as part of International Batten Disease Awareness Day, an annual effort to raise awareness about the disease. People can register to participate…

A new mutation in the MFSD8 gene was identified as the cause of CLN7 disease, a form of late-infantile Batten disease, in a young Italian girl with developmental regression and a smaller-than-normal head, a case report shows. “Our case is discussed to highlight the pivotal role of clinical genetics, and of a…

With approval from Health Canada, Taysha Gene Therapies will begin a Phase 1/2 clinical trial testing TSHA-118, its investigational gene therapy for CLN1 disease, also known as infantile Batten disease. This first trial will launch at Queen’s University, in Ontario, under the coordination of Jagdeep Walia, MD, with…

Winners of the Australia and New Zealand Batten Disease Research Grant for 2021 have been announced by the Batten Disease Support and Research Association (BDSRA) Australia, the program’s sponsor. They are Anthony White, PhD, an associate professor and principal research fellow at QIMR Berghofer Medical Research Institute in Brisbane, Australia, and Anthony…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to LX1004, an investigational gene therapy for CLN2 disease, also known as late infantile Batten disease. “Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches…