Brineura (cerliponase alfa), marketed by BioMarin Pharmaceuticals, was given the WORLDSymposium 2018 New Treatment Award in recognition of its achievements in treating lysosomal diseases, including Batten disease. Brineura slows the decline of children’s ability to crawl or walk, and is the only therapy approved by the U.S. Food and Drug…
News
A preliminary recommendation against British National Health Service insurance coverage of the $702,000-a-year therapy Brineura (cerliponase alfa) to treat a specific type of Batten disease is “cruel and inaccurately based,” the U.K. nonprofit Batten Disease Family Association contends. The expensive drug, manufactured by San Francisco-based BioMarin, is aimed…
A potential gene therapy to treat infantile Batten disease, ABO-202 by Abeona Therapeutics, has been designated an orphan drug by the U.S. Food and Drug Administration (FDA) to help advance its development in clinical testing. Also known as infantile neuronal ceroid lipofuscinosis (INCL), infantile Batten disease is a rare lysosomal storage disease…
David Curtis Glebe, a retired 64-year-old public prosecutor now living in Millsboro, Delaware, knows he’s lucky to be alive. In mid-2013, while in Arizona, Glebe was diagnosed with pancreatic neuroendocrine cancer (PNET) — the same disease that killed Apple’s founder and CEO Steve Jobs. After three years of progress…
A recent review highlights how autophagy, a process in the body that deals with the destruction of cells to maintain balance and normal functions, is impaired in patients with lysosomal storage disorders (LSDs) such as Batten disease, and could eventually be a therapeutic target for disease. The study,…
Early detection of abnormalities to the eye’s retina and loss of visual acuity may help diagnose neuronal ceroid lipofuscinosis (NCL), aka Batten disease, and improve patient care, Brazilian researchers suggest. For several neurodegenerative disorders that have symptom onset during early infancy is not uncommon for the children to develop eye…
This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…
A new bioinformatic tool called Aminode, developed to help researchers predict the potential outcome of genetic mutations, found that most disease-causing mutations occur in regions of proteins essential to their normal structure and function — including in Batten disease. In fact, nearly three-quarters of the mutations known to cause Batten…
In rare cases, a type of Batten disease may appear at birth, write researchers in a case report, describing a newborn with severe brain damage. The research team at the University Hospitals of Geneva said the disease — also called congenital neuronal ceroid lipofuscinosis type 10 (CLN10) — was…
Polaryx Therapeutics’ investigative drug PLX-100 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of neuronal ceroid lipofuscinosis (NCL), also known as Batten disease. This follows the previous announcement of ODD status granted to PLX-200, another drug being tested…
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