Risdiplam (Evrysdi, RG7916, RO7034067) is an orally administered SMN2 pre-mRNA splicing modifier that distributes into the central nervous system (CNS) and peripheral tissues.
Storage Temp
Store at -20°C
Shipped In
Ice chest + Ice pads
Grade
Moligand™
Product Description
Information
Risdiplam (RG7916) Risdiplam (Evrysdi, RG7916, RO7034067) is an orally administered SMN2 pre-mRNA splicing modifier that distributes into the central nervous system (CNS) and peripheral tissues.
1.Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, Feng Z, Green L, Guerard M et al.. (2018) Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).. J Med Chem, 61 (15):(6501-6517). [PMID:30044619]
2.Poirier A, Weetall M, Heinig K, Bucheli F, Schoenlein K, Alsenz J, Bassett S, Ullah M, Senn C, Ratni H et al.. (2018) Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.. Pharmacol Res Perspect, 6 (6):(e00447). [PMID:30519476]
3.Kletzl H, Marquet A, Günther A, Tang W, Heuberger J, Groeneveld GJ, Birkhoff W, Mercuri E, Lochmüller H, Wood C et al.. (2019) The oral splicing modifier RG7800 increases full length survival of motor neuron 2 mRNA and survival of motor neuron protein: Results from trials in healthy adults and patients with spinal muscular atrophy.. Neuromuscul Disord, 29 (1):(21-29). [PMID:30553700]
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