Batten disease is a rare genetic disorder affecting 3 in 100,000 babies in the U.S., causing neurodegeneration, seizures, ...

Parents of children with a rare neurodegenerative disease say they are in disbelief they could within months lose access to ...

She is currently treated with the drug Brineura, allowing her to attend school and live "a better quality of life". NHS ...

Analysts provide deeper insights through their assessments of 12-month price targets, revealing an average target of $94.25, a high estimate of $126.00, and a low estimate of $70.00. This current ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

BMRN reports better-than-expected fourth-quarter results, driven by encouraging uptake for Voxzogo. It reiterates its guidance of generating $4 billion in total sales by 2027.

The news was welcomed by the nine-year-old’s family, who saying stopping the medication would hasten her death.

Charleigh Pollock has a neurological disorder called Batten disease that leaves her with recurring severe seizures and ...

The mom of 9-year-old Charleigh says the clinical criteria used is outdated and 'needs to be updated immediately' ...

A Vancouver Island family continues to fight for their daughter’s life-sustaining medication, Brineura. And each birthday that Charleigh Pollock gets to celebrate is a special one.