EXCLUSIVE: NICE decision means kids diagnosed with devastating neurodegenerative condition will no longer receive life-lengthening enzyme therapy on NHS ...

The Rich family have seen first hand how much the drug can help children with a devastating Batten disease diagnosis ...

Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), joins the global ...

Michael and Lucy Carroll talk to ITV Granada Reports' Zoe Muldoon about their devastation that NICE will no longer fund a drug for Batten disease. The parents of children who are living with Batten ...

A funding decision for the sole treatment that slows the progression of Batten Disease has been made. Children diagnosed have a life expectancy between six and 12 years, writes Victoria Macdonald.

Cerliponase alfa (Brineura) is not normally available on the NHS for treating neuronal ceroid lipofuscinosis type 2 (CLN2). CLN2 is a type of Batten disease that is also known as tripeptidyl peptidase ...

rhPPT1 is a recombinant protein used to treat an ultra-rare disease called Batten disease CLN1 for which there is currently no FDA approved treatment available. These findings suggest intravenous ...