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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…
Regenxbio plans to provide updates on its RGX-181 and RGX-381 gene therapy candidates for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease, in the second half of this year. The company is currently reviewing information requests from…
Brineura (cerliponase alfa) stabilized the progression of neuronal ceroid lipofuscinosis type 2 (CLN2) — a type of Batten disease — in a small group of people in Columbia, including those with an atypical onset that occurs later in childhood.
Neurogene, a biopharmaceutical company developing therapies for Batten disease, is trying to learn more about the progression of the subtype CLN5 disease from the caregiver perspective. To that end, Neurogene is inviting the caregivers of children who are either living with CLN5, or who died within the…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…
The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…
Families of a child with Batten disease in the U.K. are welcome take part in counseling and other wellness services being offered at no cost by the Maypole Project in a three-year collaboration with the Batten Disease Family Association (BDFA). Services include one-on-one therapy sessions, as well as sessions tailored to couples,…
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…
Editor’s note: This story was updated March 30, 2021 to clarify that Neurogene is hosting videoconferences workshops for families in any country. Neurogene, which is developing treatments for Batten disease and other rare neurological disorders, is inviting families affected by CLN5, a form of late infantile…
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