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.
People with CDKL5 deficiency disorder (CDD) now have their first FDA-approved treatment, after the US regulator gave the nod to Marinus Pharma's Ztalmy for the rare genetic form of
There are approximately 300 million people around the world living with a rare disease. Yet, until relatively recently, there was limited focus on this heterogenous population.
Gene sequencing specialist Illumina has started rolling out a new test that looks for a battery of tumour-associated genes from a single tissue sample, and could be used to help guide treat
Amryt Pharma has been handed a major blow by the FDA, after the US regulator rejected its marketing application for Oleogel-S10, its drug for rare and debilitating skin disease epidermolysi
There's been hardly any change in the use of clot-dissolving (thrombolytic) therapies for conditions like heart attack and stroke for decades, but a small Dutch biotech – TargED Biopharmace