The mother of a six-year-old girl with a rare neurodegenerative disease has urged health bosses to "do the right thing" amid ...

The biotech is initially using the capsid to treat neuronal ceroid lipofuscinosis 2 (CLN2) disease. Children with the disease currently receive an enzyme-replacement therapy that entails a 4 h ...

A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare ...

A life-saving medicine for children with Batten disease, a rare and fatal genetic disorder, is set to be pulled from the market because of unresolved pricing negotiations. Otherwise called CLN2 ...

CLN2 Batten disease is an incurable genetic disorder that causes rapid decline in a child's ability to walk, talk and see. Phoebe, from Coventry, said daughter Flory's doses of Brineura ...

Parents of children with a rare neurodegenerative disease say they are in disbelief they could within months lose access to the only approved treatment that slows the condition's progress.

Also known as Batten disease, CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons ...

Vimizim in Morquio A syndrome and Brineura in CLN2 disease should also drive growth. BioMarin's highest-potential drug, Voxzogo, launched in late 2021. The drug has shown the ability to restore ...

Two years ago, Beatrice Cieslik was diagnosed with CLN2 type Batten disease - a rare degenerative condition which causes seizures, blindness, dementia, and difficulty with mobility and speech. She is ...