Ractigen receives orphan drug designation for ALS therapy
RAG-17 has been designed to act on and knockdown SOD1 expression in patients with pathogenic mutations that are known to cause ALS. The chemistry of the therapeutic is
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.