Spinal Muscular Atrophy

Risdiplam

Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA)[4][5] and the first oral medication approved to treat this disease.[4][5] Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier.[4][3][6] In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (a...

Drug Index

Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA)[4][5] and the first oral medication approved to treat this disease.[4][5] Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier.[4][3][6] In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (a...

Nusinersen

Nusinersen,[3] marketed as Spinraza,[1] is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder.[4][1] In December 2016, it became the first approved drug used in treating this disorder. Since the condition it treats is so rare, Nusinersen has so-called “orphan drug” designation in the United States and the European Union.[5] Contents 1 Medical uses 2 Side effect...

Drug Index

Nusinersen,[3] marketed as Spinraza,[1] is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder.[4][1] In December 2016, it became the first approved drug used in treating this disorder. Since the condition it treats is so rare, Nusinersen has so-called “orphan drug” designation in the United States and the European Union.[5] Contents 1 Medical uses 2 Side effect...