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New daily pill shows promise for spinal muscular atrophy

NCT ID NCT03032172

Completed Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested the safety and drug levels of risdiplam, a daily oral medication, in 174 adults and children with spinal muscular atrophy (SMA). Participants had previously received other SMA treatments. The main goals were to check for side effects and measure how the drug moves through the body. Results will help guide future use of risdiplam for SMA.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
risdiplam
What this could lead to
If it works, this could provide a daily oral treatment option for spinal muscular atrophy, potentially improving motor function and survival.
What could go wrong
This is an early-phase, open-label study without a comparison group, so results may not confirm effectiveness. Side effects and long-term risks are still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SPINAL MUSCULAR ATROPHY

Contacts and locations

Locations

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Boston Childrens Hospital

    Boston, Massachusetts, 02115, United States

  • CHRU de Montpellier, Hopital Gui de Chauliac

    Montpellier, 34295, France

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Lombardy, 20122, Italy

  • Hopital Femme Mere Enfant

    Bron, 69677, France

  • Hopital Roger Salengro

    Lille, 59037, France

  • Hopital des Enfants

    Toulouse, 31059, France

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

  • IRCCS Istituto Giannina Gaslini

    Genoa, Liguria, 16147, Italy

  • IRCCS Ospedale Pediatrico Bambino Gesù

    Rome, Lazio, 00165, Italy

  • Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie

    Warsaw, 02-097, Poland

  • Nemours Children's Hospital

    Orlando, Florida, 32827, United States

  • Policlinico Agostino Gemelli

    Rome, Lazio, 00168, Italy

  • Stanford University Medical Center

    Palo Alto, California, 94304, United States

  • The Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • UCL Institute of Child Health & Great Ormond Street Hospital for Children

    London, WC1N 1EH, United Kingdom

  • UMC Utrecht

    Utrecht, 3584 CX, Netherlands

  • UOSD Malattie Neurodegenerative

    Messina, Sicily, 98125, Italy

  • UZ Gent

    Ghent, 9000, Belgium

  • UZ Leuven Gasthuisberg

    Leuven, 3000, Belgium

  • Universitäts-Kinderspital (UKBB) Neuropädiatrie

    Basel, 4005, Switzerland

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

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