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Rarebase, a public benefit biotechnology company, has launched the Function therapeutic discovery platform specifically for rare neurological diseases, including Batten disease. Led by scientists, engineers, patients, and advocates, Function combines large-scale therapeutic screening with genetic screens, all conducted in nerve cells (neurons) grown in a dish. The combination…
An international team of experts has developed a set of consensus-based, family-informed recommendations for the diagnosis and best care management of neuronal ceroid lipofuscinosis type 1 (CLN1) disease. The guidelines were outlined in a report, “Management of CLN1 Disease: International Clinical Consensus,” published in the journal…
The Beyond Batten Disease Foundation (BBDF) is sponsoring Care Beyond Diagnosis in its efforts to draft international guidelines to help clinicians counsel and treat patients with CLN3 disease, or juvenile Batten disease. Based on clinical evidence and consensus-based recommendations, the guidelines are designed to improve patient care…
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…
A screening of U.S.-approved therapies revealed that tamoxifen, an oral cancer therapy, is able to effectively reduce the accumulation of a fatty molecule called globotriaosylceramide, or Gb3, in cellular models of two forms of Batten disease. It worked without compromising cell survival, a study showed. That study found that…
Enrollment in a clinical trial testing NGN-101 — an experimental gene therapy for CLN5, a type of late-infantile Batten disease — is expected to start early next year at the University of Rochester Medical Center (URMC). “This trial will move research forward in developing a potentially disease-modifying treatment for…
Over 230 national organizations signed a letter urging all 50 U.S. state governors to “maintain and expand” flexibility with licensure requirements for the duration of the COVID-19 pandemic to ease access to care. During the pandemic, governors used emergency authority to waive certain state licensure requirements, giving healthcare providers…
Lexeo Therapeutics announced that it raised $100 million in a financing round to help support the development of pipeline therapies, including LX1004, a potential gene therapy for CLN2 disease, also known as late infantile Batten disease. Other potential treatments supported through this Series B financing include LX2006, a gene therapy for…
A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three to five times higher than for those without rare diseases. This study provides evidence of the potential effect rare diseases may have on public health…
In their continued efforts to improve health equity for people with rare diseases, Global Genes and RARE-X have joined forces to help advocacy groups collect patient data and make the most of that information. “Patient data is perhaps the most valuable asset rare disease communities can leverage to…
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