Dr. Greg Rippon, Vice President and Chief Medical Partner of Neurology, Genentech shines light on SMA, spinal muscular atrophy, a severe inherited progressive neuromuscular disease and one of the most common rare diseases affecting babies. With the introduction of Evrysdi to the treatment options for SMA, an oral treatment is being tested to increase the production of the functional survival motor neuron protein and thus benefit patients in their motor functioning.
@genentech #genentech #SMA #SpinalMuscularAtrophy #Evrysdi #raredisease
GENE.com
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