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The enzyme replacement therapy Brineura (cerliponase alfa) is now approved by the U.S. Food and Drug Administration (FDA) as a treatment for CLN2 disease, also known as late infantile Batten disease, in children of all ages. The FDA previously approved Brineura to slow the loss of walking or crawling…
Drinking acidified laboratory water improved motor performance, restored muscle strength, prevented tremors, and extended survival in a mouse model of late infantile Batten disease, a study reports. Researchers also noted pronounced changes in gut bacterial composition in Batten mice given acidified drinking water, which might have contributed to its…
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…
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…
Note: This story was updated March 5, 2024, to clarify that Taysha Gene Therapies set aside the development of TSHA-118, along with other experimental gene therapies, to focus on another treatment for a different disorder. A patient with CLN1 disease, also known as infantile Batten disease, has received treatment…
A small molecule inhibitor of the sortilin protein reduced cellular waste product accumulation and eased neuroinflammation in mouse models of late-infantile and juvenile forms of Batten disease in a recent study. The treatment also completely eliminated tremors in a model of late-infantile…
Umbilical cord blood transplantation (UCBT) can be safely performed in children with Batten disease, a study reported. Preliminary data suggest that UCBT might be an effective treatment in some cases, particularly in people with juvenile Batten disease who are early in the course of their disease, the researchers…
An experimental gene therapy for late infantile Batten disease showed a good safety profile and appeared to be working as designed in a study done in non-human primates. “The advancement of this potential gene therapy could provide new hope to families with affected children,” Terence R. Flotte, MD, editor-in-chief…
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…
Managing fear episodes in people with juvenile Batten disease requires minimizing situations that provoke patients, and increasing their threshold of pain and discomfort, a study reports. Sedative medications and therapies that activate specific areas of the nervous system may also help manage such responses, but solid evidence for them…
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