SMA THERAPIES II AND BIOMARKERS: P.255SUNFISH Part 1: RG7916 treatment results in a sustained increase of SMN protein levels and the first clinical efficacy results in patients with type 2 or 3 SMA.

Authors :: Mercuri, E.
Baranello, G.
Kirschner, J.
Servais, L.
Goemans, N.
Carmela Pera, M.
Buchbjerg, J.
Armstrong, G.
Kletzl, H.
Gerber, M.
Czech, C.
Cleary, Y.
Gorni, K.
Khwaja, O.
Source :: Neuromuscular Disorders. Oct2018:Supplement 2, Vol. 28, pS108-S108. 1p.
Publication Year :: 2018

Subjects :: *TREATMENT of spinal muscular atrophy
*SPINAL muscular atrophy
*CLINICAL trials
*PATIENTS

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