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Hope for the youngest: testing SMA drug in Babies' first days

NCT ID NCT05808764

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

Summary

This study aims to understand how the drug risdiplam works in the bodies of very young infants with spinal muscular atrophy (SMA). It will enroll up to 10 newborns under 20 days old to measure the drug's levels in their blood and check for safety. The goal is to gather information to potentially help control this serious muscle-wasting disease as early as possible.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ann and Robert H. Lurie Children Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • CHR Citadelle

    RECRUITING

    Liège, 4000, Belgium

  • Children'S Hospital of Eastern Ontario

    RECRUITING

    Ottawa, Ontario, K1H 8L1, Canada

  • Clinic for Special Children.

    RECRUITING

    Gordonville, Pennsylvania, 17529, United States

  • Fondazione Policlinico Univeristario A. Gemelli

    RECRUITING

    ROMA, Emilia-Romagna, 00168, Italy

  • Fondazione Serena Onlus - CENTRO CLINICO NEMO

    RECRUITING

    Milano, Emilia-Romagna, 20162, Italy

  • Hopital Universitaire des Enfants Reine Fabiola

    RECRUITING

    Brussels, 1020, Belgium

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    RECRUITING

    Warsaw, 04-730, Poland

  • OUS (Oslo University Hospital), Rikshospitalet

    RECRUITING

    Oslo, 0372, Norway

  • UMC Utrecht

    RECRUITING

    Utrecht, 3508, Netherlands

  • Universitatsklinikum Essen

    RECRUITING

    Essen, 45147, Germany

  • University Of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Uniwersyteckie Centrum Kliniczne

    RECRUITING

    Gdansk, 80-952, Poland

Conditions

Explore the condition pages connected to this study.

MUSCULAR ATROPHY, SPINAL