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Re: "Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy" by Wilson and Flotte

Shieh, P. B.; Bönnemann, C. G.; Müller-Felber, W.; Blaschek, A.; Dowling, J. J.; Kuntz, N. L.; Seferian, A. M. Hum Gene Ther. 2020 Aug 12; 31(15-16):787

Combination molecular therapies for type 1 spinal muscular atrophy

Harada, Y.; Rao, V. K.; Arya, K.; Kuntz, N. L.; DiDonato, C. J.; Napchan-Pomerantz, G.; Agarwal, A.; Stefans, V.; Katsuno, M.; Veerapandiyan, A. Muscle Nerve. 2020 Jul 28

The CINRG Becker Natural History Study: Baseline characteristics

Clemens, P. R.; Niizawa, G.; Feng, J.; Florence, J.; DʼAlessandro, A. S.; Morgenroth, L. P.; Gorni, K.; Guglieri, M.; Connolly, A.; Wicklund, M.; Bertorini, T.; Mah, J. K.; Thangarajh, M.; Smith, E.; Kuntz, N.; McDonald, C. M.; Henricson, E. K.; Upadhyayula, S.; Byrne, B.; Manousakis, G.; Harper, A.; Bravver, E.; Iannaccone, S.; Spurney, C.; Cnaan, A.; Gordish-Dressman, H. Muscle Nerve. 2020 Jun 22; 62(3):369-376

Spinal muscular atrophy: Nature or nurture?

Topaloglu, H.; Kuntz, N. Neurology. 2020 Jun 10; 95(1):11-12

Difficulties classifying myasthenia gravis in the pediatric surgical literature

Goldstein, S. D.; Kuntz, N. L. J Pediatr Surg. 2020 May 24; 55(8):1679