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Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies
Oral SMN2 splicing modifiers in spinal muscular atrophy: Proof-of-mechanism and ongoing clinical studies
2017
Anne Marquet
Stefan Sturm
Heidemarie Kletzl
Andreas Günther
Christian Czech
Agnès Poirier
Timothy Seabrook
Lutz Müller
Russell Kinch
Hasane Ratni
K. Gorni
Friedrich Metzger
Omar Khwaja
Keywords:
Spinal muscular atrophy
Diabetes mellitus
RNA splicing
Medicine
Bioinformatics
Physical therapy
Correction
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