Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs.
Poirier, Agnès
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. [electronic resource] - Pharmacology research & perspectives 12 2018 - e00447 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
2052-1707
10.1002/prp2.447 doi
Animals
Azo Compounds--cerebrospinal fluid
Brain--metabolism
Clinical Trials as Topic
Disease Models, Animal
Dogs
Drug Evaluation, Preclinical
Exons--drug effects
Female
Humans
Macaca fascicularis
Madin Darby Canine Kidney Cells
Male
Mice
Muscular Atrophy, Spinal--drug therapy
Neuromuscular Agents--cerebrospinal fluid
Pyrimidines--cerebrospinal fluid
RNA Splicing--drug effects
Rats
Rats, Wistar
Survival of Motor Neuron 1 Protein--metabolism
Survival of Motor Neuron 2 Protein--genetics
Swine
Tissue Distribution
Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. [electronic resource] - Pharmacology research & perspectives 12 2018 - e00447 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
2052-1707
10.1002/prp2.447 doi
Animals
Azo Compounds--cerebrospinal fluid
Brain--metabolism
Clinical Trials as Topic
Disease Models, Animal
Dogs
Drug Evaluation, Preclinical
Exons--drug effects
Female
Humans
Macaca fascicularis
Madin Darby Canine Kidney Cells
Male
Mice
Muscular Atrophy, Spinal--drug therapy
Neuromuscular Agents--cerebrospinal fluid
Pyrimidines--cerebrospinal fluid
RNA Splicing--drug effects
Rats
Rats, Wistar
Survival of Motor Neuron 1 Protein--metabolism
Survival of Motor Neuron 2 Protein--genetics
Swine
Tissue Distribution