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The New England Journal of Medicine|July 5, 2018
Inotersen Treatment for Patients with Hereditary Transthyretin AmyloidosisMerrill D Benson, Márcia Waddington-Cruz, John L Berk, et al.The New England Journal of Medicine|July 5, 2018
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin AmyloidosisDavid Adams, Alejandra Gonzalez-Duarte, William D O'Riordan, et al.The New England Journal of Medicine|September 26, 2019
Step-Up Therapy in Black Children and Adults with Poorly Controlled AsthmaMichael E Wechsler, Stanley J Szefler, Victor E Ortega, et al.Orphanet Journal of Rare Diseases|October 27, 2021
A multicenter cross-sectional French study of the impact of COVID-19 on neuromuscular diseasesLucie Isoline Pisella, Sara Fernandes, Guilhem Solé, et al.The New England Journal of Medicine|April 11, 2014
Glycosylation, hypogammaglobulinemia, and resistance to viral infectionsMohammed A Sadat, Susan Moir, Tae-Wook Chun, et al.Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology|March 14, 2020
Factors Affecting Sentinel Node Metastasis in Thin (T1) Cutaneous Melanomas: Development and External Validation of a Predictive NomogramAndrea Maurichi, Rosalba Miceli, Hanna Eriksson, et al.Investigative Ophthalmology & Visual Science|May 5, 2025
Systematic Ocular Phenotyping of Knockout Mouse Lines Identifies Genes Associated With Age-Related Corneal DystrophiesAndrew Briere, Peter Vo, Benjamin Yang, et al.Journal of Clinical Immunology|September 23, 2024
Biallelic PI4KA Mutations Disrupt B-Cell Metabolism and Cause B-Cell Lymphopenia and HypogammaglobulinemiaFrancesco Saettini, Fabiola Guerra, Mario Mauri, et al.American Journal of Human Genetics|October 6, 2018
A Recurrent De Novo Heterozygous COG4 Substitution Leads to Saul-Wilson Syndrome, Disrupted Vesicular Trafficking, and Altered Proteoglycan GlycosylationCarlos R Ferreira, Zhi-Jie Xia, Aurélie Clément, et al.JAMA|July 30, 2025
Influenza-Associated Acute Necrotizing Encephalopathy in US Children, Andrew Silverman, Rachel Walsh, et al.Pageof 34