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Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.
Nature Medicine|June 17, 2022
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trialKevin A Strauss, Michelle A Farrar, Francesco Muntoni, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|May 13, 2016
Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York StateMelissa P Wasserstein, Mary Andriola, Georgianne Arnold, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|January 23, 2016
Newborn screening for Krabbe disease in New York State: the first eight years' experienceJoseph J Orsini, Denise M Kay, Carlos A Saavedra-Matiz, et al.
The Lancet. Neurology|March 20, 2021
Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trialJohn W Day, Richard S Finkel, Claudia A Chiriboga, et al.
Pediatric Neurology|March 24, 2009
Newborn screening for Krabbe disease: the New York State modelPatricia K Duffner, Michele Caggana, Joseph J Orsini, et al.
The Journal of Allergy and Clinical Immunology. in Practice|July 13, 2024
Interleukin (IL)-1/IL-6-Inhibitor-Associated Drug Reaction With Eosinophilia and Systemic Symptoms (DReSS) in Systemic Inflammatory IllnessesVivian E Saper, Lu Tian, Ruud H J Verstegen, et al.
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