Stephen B. Heitner

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

Merrill D. Benson, Márcia Waddington‐Cruz, John L. Berk et al. · New England Journal of Medicine · 2018 · 1.4K citations · Full text

Life Sciences, Pharmacology, Pharmacotherapy +16

Correction of a pathogenic gene mutation in human embryos

Hong Ma, Nuria Martí‐Gutiérrez, Sang-Wook Park et al. · Nature · 2017 · 951 citations

Life Sciences, Developmental Biology, Genetics +12

Common genetic variants and modifiable risk factors underpin hypertrophic cardiomyopathy susceptibility and expressivity

Andrew R. Harper, Anuj Goel, Christopher Grace et al. · Nature Genetics · 2021 · 339 citations · Full text

Medicine, Genetics, Molecular Genetics +14

Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy

Carolyn Y. Ho, Matthew E. Mealiffe, Richard G. Bach et al. · Journal of the American College of Cardiology · 2020 · 338 citations · Full text

Medicine, Cardiology, Pharmacology +14

Distinct Subgroups in Hypertrophic Cardiomyopathy in the NHLBI HCM Registry

Stefan Neubauer, Paul Kolm, Carolyn Y. Ho et al. · Journal of the American College of Cardiology · 2019 · 321 citations · Full text

Medicine, Cardiology, Clinical Cardiology +1