In 2015, NICE said that BioMarin's Vimizim could be used to treat patients with the ultra-rare disease Morquio A syndrome, while real-world data was collected on its efficacy.
NICE says it can approve BioMarin's ultra-rare drug treatment Vimizim, but has proposed real-world data collection and a price cap to overcome doubts about the drug's value, reports Andrew McCo
Software that can be used to monitor patients with neurodegenerative disorder amyotrophic lateral sclerosis (ALS) using their voice patterns has been granted breakthrough