Sentences with phrase «muscular atrophy focus»
Nusinersen and similar compounds being developed for spinal muscular atrophy focus on increasing or making more of the SMN gene product that gets converted into usable protein.
https://www.sciencedaily.com/ Not exact matches
It's aggressively focused on curing spinal muscular atrophy.
SOUTH PLAINFIELD, NJ — December 6, 2007 — PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small - molecule drugs targeting post-transcriptional control mechanisms, today announced an expanded research collaboration with the Spinal Muscular Atrophy (SMA) Foundation.