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Treatment Effects Among Patients with Type 2 and Type 3 SMA Directly Reported by Patients and Caregivers from the TOPAZ Clinical Trial

Author:Pokrzywinski, Robin   Cutts, Katelyn   Shah, Hemal   Kertesz, Nathalie   Lesh, Sabrina   Sadanowicz, Mara   Song, Guochen   O'Neil, Janet   Barrett, Doreen   Barnobi, Erin   Baver, Scott   

Session Name:P2: Neuromuscular Disease: ALS, SMA, and Other Motor Neuron Disorders 1  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:P2.010  

Author Institution:Patient-Centered Research, Evidera, Bethesda, MD  alue Matters, LLC. New York, NY  Scholar Rock, Inc. Cambridge, MA