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An investigational gene therapy for late infantile (CLN2) Batten disease showed a good safety profile in a small clinical trial. The gene therapy also slowed the progression of the disease, though not to the extent of current standard-of-care treatment. Researchers are now working on more effective ways to deliver…
Biopharmaceutical company Theranexus has reached an exclusive $20 million global agreement with Beyond Batten Disease Foundation (BBDF) for an upcoming clinical trial and potential commercialization of juvenile Batten disease treatment candidate BBDF-101. The pact follows a June 27 agreement in principle, in which Theranexus was granted six…
New research has deepened the understanding of the underlying causes of Batten disease, including organs and cell types affected as well as crucial molecular mechanisms, which can help the design of novel therapies, a review study reports. Researchers believe these new insights will be key to inform the targeting,…
Those afflicted with lysosomal storage disorders such as Batten disease may benefit from a clever new treatment strategy one day. A recent study has demonstrated how to turn a certain antibody into a so-called Trojan horse for ferrying healthy enzymes missing in these disorders across the blood-brain barrier. This…
Drinking acidified water may have beneficial effects for children with CLN3 disease, the juvenile form of Batten disease, a study performed in a mouse model suggests. The effects of water with higher acid levels are probably linked to changes in the population of microorganisms that live in the…
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
Sanger sequencing — a conventional genetic test useful to identify mutations in disease-associated genes — can effectively help to provide a definitive diagnosis of Batten disease, according to a Canadian study. The research also highlighted that the presence of cognitive decline, loss of previously acquired skills, progressive visual problems, and…
CLN7, the protein lacking in CLN7 Batten disease, is involved not only in the functioning of lysosomes (the cellular compartment responsible for breaking down waste) but also in the communication between nerve and muscle cells, according to a preclinical study. These findings open the possibility that proteins associated with other…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
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