Higher dose of SMA drug shows promise for those who switched treatments

NCT ID NCT05067790

First seen May 25, 2026 · Last updated May 25, 2026

Summary

This study looks at whether a higher dose of nusinersen can improve arm and hand function in teenagers and adults with spinal muscular atrophy (SMA) who cannot walk and have already taken another SMA drug called risdiplam. Participants receive two high loading doses, then regular maintenance doses over about 2 years, with an option to continue for another 2 years. The main goal is to measure changes in upper limb movement using a standard test.

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Contacts and locations

Locations

  • Barrow Neurological Institute

    Phoenix, Arizona, 85013, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115-5724, United States

  • Columbia University

    New York, New York, 10032, United States

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Fondazione Serena Onlus - Centro Clinico Nemo

    Milan, 20162, Italy

  • Klinikum rechts der Isar der TU Muenchen

    Munich, Bavaria, 81675, Germany

  • Memorial Healthcare

    Owosso, Michigan, 48867, United States

  • NHO Osaka Toneyama Medical Center

    Toyonaka-shi, Osaka, 560-8552, Japan

  • Neurology Rare Disease Center

    Denton, Texas, 76208, United States

  • Ospedale S G Battista Molinette

    Torino, 10126, Italy

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Semmelweis Egyetem

    Budapest, 1085, Hungary

  • Stanford Neuroscience Health Center

    Palo Alto, California, 94304, United States

  • The University of Texas Health Science Center at Houston

    Houston, Texas, 77030, United States

  • Universitaetsklinikum Essen

    Essen, North Rhine-Westphalia, 45122, Germany

  • Universitaetsklinikum Heidelberg

    Heidelberg, Baden-Wurttemberg, 69120, Germany

  • Universitaetsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Washington Medical Center

    Seattle, Washington, 98195, United States

  • Wake Forest University - School of Medicine - Central

    Winston-Salem, North Carolina, 27157, United States

  • Yokohama City University Hospital

    Yokohama, Kanagawa, 236-0004, Japan

Conditions

Explore the condition pages connected to this study.