Entries by English RSS Feed

Shaping the future of sickle cell disease

Majara, a 32-year-old woman from Greenville, North Carolina, in the US, vividly remembers the times when her sickle cell pain crises gripped her body so badly she couldn’t move. “It felt like a dump truck had actually fallen on my body and was just sit…

AVXS-101 Managed Access Program

StatementFeb 05, 2020We are aware of the recent Ministry of Health (MoH) announcement, that five EU states (Belgium, the Netherlands, Luxembourg, Austria and Ireland) have issued a joint statement regarding our Global Managed Access Program (MAP).  In …