TY  - GEN
AU  - Poirier,Agnès
AU  - Weetall,Marla
AU  - Heinig,Katja
AU  - Bucheli,Franz
AU  - Schoenlein,Kerstin
AU  - Alsenz,Jochem
AU  - Bassett,Simon
AU  - Ullah,Mohammed
AU  - Senn,Claudia
AU  - Ratni,Hasane
AU  - Naryshkin,Nikolai
AU  - Paushkin,Sergey
AU  - Mueller,Lutz
TI  - Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
SN  - 2052-1707
PY  - 2019///0204
KW  - Animals
KW  - Azo Compounds
KW  - cerebrospinal fluid
KW  - Brain
KW  - metabolism
KW  - Clinical Trials as Topic
KW  - Disease Models, Animal
KW  - Dogs
KW  - Drug Evaluation, Preclinical
KW  - Exons
KW  - drug effects
KW  - Female
KW  - Humans
KW  - Macaca fascicularis
KW  - Madin Darby Canine Kidney Cells
KW  - Male
KW  - Mice
KW  - Muscular Atrophy, Spinal
KW  - drug therapy
KW  - Neuromuscular Agents
KW  - Pyrimidines
KW  - RNA Splicing
KW  - Rats
KW  - Rats, Wistar
KW  - Survival of Motor Neuron 1 Protein
KW  - Survival of Motor Neuron 2 Protein
KW  - genetics
KW  - Swine
KW  - Tissue Distribution
N1  - Publication Type: Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1002/prp2.447
ER  -