News

Recommendation Against U.K. Coverage of Brineura Jolts Batten Disease Community

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…

Early Detection of Retinal Dysfunction May Help Diagnosis

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…

FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

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…