Suchergebnisse - "Shieh, Perry B"

PubPharm (116)

41

Reldesemtiv in Patients with Spinal Muscular Atrophy: a Phase 2 Hypothesis-Generating Study

enthalten in: NeuroRX | 2021

von Rudnicki, S. | Andrews, J. | Duong, T. | +20

42

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping : PROMOVI Trial

enthalten in: Journal of neuromuscular diseases | 2021

von McDonald, C. | Shieh, P. | Abdel-Hamid, H. | +12

43

Safety and efficacy of nusinersen in spinal muscular atrophy : The EMBRACE study

enthalten in: Muscle & Nerve | 2021

von Acsadi, G. | Crawford, T. | Müller‐Felber, W. | +8

44

Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon45 skipping : A randomized, double‐blind, placebo‐controlled, dose‐titration trial

enthalten in: Muscle & Nerve | 2021

von Wagner, K. | Kuntz, N. | Koenig, E. | +4

45

Long‐term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis

enthalten in: Muscle & Nerve | 2021

von Sansone, V. | Johnson, N. | Hanna, M. | +5

46

Incorporating Spinal Muscular Atrophy Analysis by Next-Generation Sequencing into a Comprehensive Multigene Panel for Neuromuscular Disorders

enthalten in: Genetic testing and molecular biomarkers | 2020

von Tan, C. | Westbrook, M. | Truty, R. | +4

47

Congenital myasthenic syndrome caused by a frameshift insertion mutation in GFPT1

enthalten in: Neurology. Genetics | 2020

von Szelinger, S. | Krate, J. | Ramsey, K. | +20

48

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

enthalten in: Human gene therapy | 2020

von Shieh, P. | Bönnemann, C. | Müller-Felber, W. | +4

CommentOn: Hum Gene Ther. 2020 Jul;31(13-14):695-696. - PMID 32605399

49

Advances in the Genetic Testing of Neuromuscular Diseases

enthalten in: Neurologic clinics | 2020

von Shieh, P.

50

The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic

enthalten in: Muscle & nerve | 2020

von Veerapandiyan, A. | Wagner, K. | Apkon, S. | +12