Sobi’s metabolic disorder drug secures fast track status in US
MPS IIIA, a rare metabolic disorder also known as Sanfilippo syndrome type A, affects children from an early age. The condition results in severe deterioration of the central nervous
Ionis Pharmaceuticals has entered a licence agreement granting global pharmaceutical company Recordati exclusive rights to develop and commercialise zilganersen to treat Alexander disease (AxD) in countries outside the US.
SymBio holds approval for TREAKISYM injectables which are already used for the treatment of three indications of malignant lymphoma (first-line and relapsed/refractory low-grade B-cell non-Hodgkin's lymphoma and mantle