Potent IRAK-4 inhibitors have been reported by several groups including some structurally related benzimidazoles as well as alternative fused heterocycles such as the imidazopyridine and imidazopyridazine. Such compounds are reported to have IRAK-1 and IRAK-4 activity with varying degrees of selectivity.
As the first approved small molecule splicing modifier drug, Risdiplam (Evrysdi) is granted approval by the European Commission to expand the EU marketing authorization. This extension now includes infants with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months. Type 1 spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease and a leading genetic factor of infant mortality, that is caused by low levels of functional survival of motor neuron (SMN) protein. Risdiplam modifies SMN2 pre-messenger RNA splicing and increases levels of functional SMN protein in both the central nervous system and peripheral organs.
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