Publications by authors named "R C Finkel"

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Exposure-response of serum biomarkers to vamorolone, a dissociative corticosteroidal anti-inflammatory drug, in 4- to <7-year children.
Swati Mummidivarpu, Utkarsh J Dang, Michael Ziemba, Yetrib Hathout, Paula R Clemens, Richard S Finkel

Steroids· January 2026

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Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.
Crystal M Proud, Dũng Chí Vũ, Jo M Wilmshurst, Oranee Sanmaneechai, Sheffali Gulati, Richard S Finkel

Nat Med· December 2025

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Respiratory management in spinal muscular atrophy: development of a global outcome measure.
L Edel, M Civitello, F Muntoni, R Finkel, E Mercuri

Neuromuscul Disord· November 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, Richard S Finkel

Expert Rev Neurother· November 2025

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Ad astra per aspera: treatment challenges and opportunities for children with spinal muscular atrophy and tracheostomy.
Yasemin Erbas, Richard S Finkel

Gene Ther· November 2025

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