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The stability of lysosomal proteins when tested in a laboratory setting is not equivalent to that observed in live cells, a study has found. This information should be considered when designing new protein versions to improve the effectiveness of enzyme replacement therapy for Batten disease. Results of…
Children with rapid and progressive vision loss — regardless of cognitive or behavioral problems — should be tested for CLN3 disease, the juvenile form of Batten disease, a study suggests. The researchers pointed out that blood film microscopy (where a blood drop is analyzed under microscope) is a fast, accessible,…
Neurogene, which is developing therapies to treat people with Batten disease, has joined with other companies in supporting and expanding Behind the Seizure, a collaborative effort to provide free genetic testing to children in the U.S. and Canada with…
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.
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