Vapromin®
INN: risdiplam
Risdiplam is a splicing modifier of the survival motor neuron 2 (SMN2) gene messenger ribonucleic acid (pre-mRNA) precursor. Risdiplam corrects SMN2 splicing and increases the amount of functional SMN protein. It prevents the progression of SMA and helps stabilize and/or improve motor function in patients.
Vapromin® is a drug for the pathogenetic treatment of spinal muscular atrophy (SMA) in adults and children.
Spinal muscular atrophy (SMA) is a hereditary disorder caused by a mutation on chromosome 5q, resulting in a deficiency of the survival motor neuron protein (SMN). SMN ensures the function of spinal motor neurons, which transmit impulses to muscles, causing them to contract. A lack of SMN protein leads to muscle weakness and atrophy.