With more than 6,000 different diseases comprising the rare disease (RD) landscape, connecting patients and practitioners with the expertise needed to achieve th
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
