Nitrosamines can be formed through various chemical processes, including the reaction between nitrites and secondary amines in certain conditions. It is a N-Nitroso of secondary amine present in Risdiplam. Nitrosamines are part of a broader class of compounds known as N-nitroso compounds.
Risdiplam is a medication used for the treatment of spinal muscular atrophy (SMA), a genetic neuromuscular disorder. Structurally, risdiplam is a small molecule that modifies the splicing of the survival motor neuron 2 (SMN2) gene, promoting the production of functional SMN protein. This is essential for motor neuron survival and function. By increasing SMN protein levels, risdiplam helps alleviate the symptoms of SMA, including muscle weakness and atrophy. Administered orally, risdiplam offers a convenient treatment option for both pediatric and adult patients with SMA. Its use is typically guided by healthcare professionals based on individual patient characteristics and the specific type of SMA. Risdiplam represents a significant advancement in the therapeutic landscape for SMA, providing a targeted approach to address the underlying genetic cause of the disorder.
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