The US Food and Drug Administration (FDA) rejected Akcea and Ionis’ application for the use of Waylivra in treatment of familial chylomicronemia syndrome (FCS) despite earlier support of ex
It’s common for patients to want to know more about what’s causing them to be ill and their DMD diagnosis and it’s the same for their families – however long that road may be.
Maryse Roger has firsthand experience of Leber’s hereditary optic neuropathy (LHON), having first assisted her two brothers as their sight deteriorated and then leading the care of her son
