Publications by authors named "Basil Darras"

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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Jennifer M Kwon, Francina Munell, Laure Le Goff, Kotaro Yuge, Tamaki Kato, Basil T Darras

Nat Med· December 2025

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A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy.
Thomas O Crawford, Laurent Servais, Eugenio Mercuri, Heike Kölbel, Nancy Kuntz, Basil T Darras

Expert Rev Neurother· November 2025

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Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration.
Katlyn Elizabeth McGrattan, Alicia Hofelich Mohr, Anna Miles, Jacqui Allen, Juliet Ochura, Basil T Darras

Orphanet J Rare Dis· October 2025

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Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial.
Thomas O Crawford, Laurent Servais, Eugenio Mercuri, Heike Kölbel, Nancy Kuntz, Basil T Darras

Lancet Neurol· September 2025

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Retrospective Cohort Analysis of Clinical, Molecular, and Histopathologic Characteristics of 275 Patients With Nemaline Myopathy.
Clara Hildebrandt, Casie A Genetti, Tanya Logvinenko, Wathone Win, Pamela Barraza-Flores, Basil T Darras

Neurol Genet· August 2025

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Longitudinal Assessment of 4-Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen.
Giorgia Coratti, Francesca Bovis, Marika Pane, Amy Pasternak, Emilio Albamonte, Basil T Darras

Eur J Neurol· July 2025

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Spinal Muscular Atrophy Functional Composite Score Revised (SMA-FCR) in Untreated and Nusinersen-Treated Patient Cohorts.
Amy Pasternak, Michael P McDermott, Jacqueline Montes, Allan M Glanzman, Giorgia Coratti, Basil T Darras

Neurology· July 2025

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Further Comments on Risdiplam for Prenatal Therapy of Spinal Muscular Atrophy.
Crystal J J Yeo, Darryl C De Vivo, Basil T Darras

N Engl J Med· July 2025

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Characterization of swallowing biomechanics and function in untreated infants with spinal muscular atrophy: A natural history dataset.
Katlyn Elizabeth McGrattan, Robert J Graham, Alicia Hofelich Mohr, Anna Miles, Jacqui Allen, Basil T Darras

J Neuromuscul Dis· May 2025

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Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1.
Preeti Kumari, Lauren M Sullivan, Zhaozhi Li, E Parker Conquest, Elizabeth Cornforth, Basil T Darras

Nat Commun· March 2025

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Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations.
Mary K Schroth, Jennifer Deans, Diana X Bharucha Goebel, W Bryan Burnette, Basil T Darras

Neurol Clin Pract· February 2025

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Exome and Genome Sequencing to Diagnose the Genetic Basis of Neonatal Hypotonia: An International Consortium Study.
Sarah U Morton, Gregory Costain, Courtney E French, Emma Wakeling, Anna Szuto, Basil T Darras

Neurology· January 2025

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Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study.
Hugh J McMillan, Giovanni Baranello, Michelle A Farrar, Craig M Zaidman, Teresa Moreno, Basil T Darras

Neurology· January 2025

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Long-term natural history in type II and III spinal muscular atrophy: a 4-year international study on the Hammersmith Functional Motor Scale Expanded.
Giorgia Coratti, Francesca Bovis, Maria Carmela Pera, Matthew Civitello, Annemarie Rohwer, Basil T Darras

Eur J Neurol· December 2024

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Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module.
Giorgia Coratti, Matthew Civitello, Annemarie Rohwer, Emilio Albamonte, Jacqueline Montes, Basil T Darras

Neuromuscul Disord· November 2024

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Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.
Giorgia Coratti, Francesca Bovis, Maria Carmela Pera, Mariacristina Scoto, Jacqueline Montes, Basil T Darras

Eur J Neurol· August 2024

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Changes in abilities over the initial 12 months of nusinersen treatment for type II SMA.
Giorgia Coratti, Matthew Civitello, Annemarie Rohwer, Francesca Salmin, Allan M Glanzman, Basil T Darras

Neuromuscul Disord· August 2024

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Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls.
Joel Iff, Nicolae Done, Edward Tuttle, Yi Zhong, Fangzhou Wei, Basil T Darras

Muscle Nerve· July 2024

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Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.
Thomas O Crawford, John W Day, Darryl C De Vivo, Jena M Krueger, Eugenio Mercuri, Basil T Darras

Front Neurol· July 2024

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Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3.
Federica Trucco, Deborah Ridout, Harriet Weststrate, Mariacristina Scoto, Annemarie Rohwer, Basil T Darras

Neurol Clin Pract· June 2024

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Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.
Amy Wolfe, Georgia Stimpson, Danielle Ramsey, Giorgia Coratti, Sally Dunaway Young, Basil T Darras

J Neuromuscul Dis· May 2024

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Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy.
Damien Meng-Kiat Leow, Yang Kai Ng, Loo Chien Wang, Hiromi Wl Koh, Tianyun Zhao, Basil T Darras

J Clin Invest· May 2024

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Neuromuscular problems of the critically Ill neonate and child.
Leslie H Hayes, Basil T Darras

Semin Pediatr Neurol· April 2024

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Beyond Contractures in Spinal Muscular Atrophy: Identifying Lower-Limb Joint Hypermobility.
Elizabeth R Harding, Cara H Kanner, Amy Pasternak, Allan M Glanzman, Sally Dunaway Young, Basil T Darras

J Clin Med· April 2024

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Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3: The Phase 2 TOPAZ Study.
Thomas O Crawford, Basil T Darras, John W Day, Sally Dunaway Young, Tina Duong

Neurology· March 2024

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