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Muscle & Nerve|April 8, 2015
Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophyJacqueline Montes, Allan M Glanzman, Elena S Mazzone, et al.
The New England Journal of Medicine|November 2, 2017
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular AtrophyRichard S Finkel, Eugenio Mercuri, Basil T Darras, et al.
Molecular Cancer Therapeutics|July 5, 2021
Phase 0 Clinical Trial of Everolimus in Patients with Vestibular Schwannoma or MeningiomaMatthias A Karajannis, Audrey Mauguen, Ekrem Maloku, et al.
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.
The New England Journal of Medicine|February 15, 2018
Nusinersen versus Sham Control in Later-Onset Spinal Muscular AtrophyEugenio Mercuri, Basil T Darras, Claudia A Chiriboga, 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.
Contemporary Clinical Trials Communications|August 11, 2018
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!Amy Bartlett, Stephen J Kolb, Allison Kingsley, et al.
Cancer Immunology Research|August 31, 2021
Targeting the Atf7ip-Setdb1 Complex Augments Antitumor Immunity by Boosting Tumor ImmunogenicityHai Hu, Alireza Khodadadi-Jamayran, Igor Dolgalev, et al.
Blood Cancer Discovery|May 20, 2026
RAS pathway activation and microenvironmental adaptation as hallmarks of myeloid sarcomaBettina Nadorp, Audrey Lasry, Sanam Loghavi, et al.
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