SMA THERAPIES II AND BIOMARKERS: P.257JEWELFISH: RG7916 increases SMN protein in patients with SMA that have previously received therapies targeting SMN2 splicing.

Authors :: Chiriboga, C.
Mercuri, E.
Fischer, D.
Kraus, D.
Thompson, N.
Armstrong, G.
Kletzl, H.
Gerber, M.
Cleary, Y.
Bergauer, T.
Gorni, K.
Khwaja, O.
Source :: Neuromuscular Disorders. Oct2018:Supplement 2, Vol. 28, pS109-S109. 1p.
Publication Year :: 2018

Subjects :: *TREATMENT of spinal muscular atrophy
*SPINAL muscular atrophy
*RNA splicing
*PATIENTS

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