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The European Medicines Agency (EMA) designated Neurogene’s gene therapy candidate for CLN5, a form of late-infantile Batten disease, an orphan drug as a potential treatment of the disorder. “This regulatory designation is an important milestone toward addressing the urgent, unmet need of families and patients living with CLN5, and…
A gene therapy designed to deliver a working version of the CLN8 gene — the one mutated in people with CLN8, a form of late-infantile Batten disease — prolonged the survival, reduced disease-associated brain features, and partially corrected motor problems in a mouse model. The findings, likely representing one of…
To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
Two years of treatment with Brineura (cerliponase alfa) safely and effectively delayed the onset of late-infantile Batten disease in a 23-month-old boy who had not exhibited symptoms when he started treatment, a case study shows. The same treatment slowed disease progression in his older sister, who received the therapy…
Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…
From participating in a virtual 5K to sharing family stories and videos, supporters are gearing up for International Batten Disease Awareness Day on June 9. The goal is to heighten awareness among the general public as well as  lawmakers, public authorities, industry representatives, scientists, and health professionals. Awareness and education…
An international team of experts has provided the latest evidence-based recommendations for diagnosis, assessment, management, and treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a study reported. The study, “Guidelines on the diagnosis, clinical assessments, treatment and management for…
The Batten Disease Support and Research Association (BDSRA) Australia has announced the winners of the 2020 Batten disease research grant program in Australia and New Zealand. The purpose of the program is to support research in disease mechanisms, novel therapies, improvements in diagnosis, and treatment and disease management in Batten…
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