Pamela Bassett, DMD, MBA

Small Molecules Taking the Lead – Starting PTC Therapeutics (PTCT) Ahead of Key Data Catalysts 1H21

Recommendation Summary PTC Therapeutics (NASDAQ: PTCT, $57.23, BUY) is a rapidly emerging commercial-phase company focused primarily on neuromuscular and rare diseases, that we think has reached an inflection point, enabling and ensuring near- and longer-term growth. The company is proving its leadership position in small molecule RNA-focused drugs with its second product launched, Evrysdi (risdiplam), an RNA splice-modifying small-molecule to treat Spinal Muscular Atrophy (SMA). The company’s first RNA-focused small molecule, Translarna (ataluren), increases expression

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