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FDA Approves New SMA Treatment: A Major Milestone for the Community

Home / TREAT-NMD News / FDA Approves New SMA Treatment: A Major Milestone for the Community

FDA Approves New SMA Treatment: A Major Milestone for the Community

We are thrilled to share an important development for the spinal muscular atrophy (SMA) community. The U.S. Food and Drug Administration (FDA) has approved Novartis’ Itvisma® offering a new treatment option for children aged 2 and older, as well as teens and adults living with SMA.

This approval marks a significant step forward, reflecting decades of research, innovation, and collaboration within the neuromuscular field. Itvisma expands therapeutic choices for families and individuals affected by SMA, reinforcing the progress being made toward improving care and outcomes.

Read the press release here: https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-itvisma-only-gene-replacement-therapy-children-two-years-and-older-teens-and-adults-spinal-muscular-atrophy-sma

27 November 2025

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  • Home
  • Who We Are
    • About Us
    • Partnerships and Collaborations
    • Our Team
  • Who We Support
    • Patients
    • Clinicians / Researchers
    • Life Sciences Industry
  • What We Do
    • Advisory Committee for Therapeutics
      • Members of the Advisory Committee for Therapeutics
      • Take Part in TACT
      • Past Applicants
    • The Global Registry Network
      • Members of the Registry Network
    • Core Datasets
      • DM dataset
      • DMD dataset
      • FSHD dataset
      • LGMD
      • SMA
    • Education
      • Masterclasses and Events
      • Digital Resources
      • Endorsement of External Programmes
    • Post-Marketing Surveillance
  • Resources and Support
    • Neuromuscular Disease Information
      • Becker muscular dystrophy
      • Charcot-Marie-Tooth
      • Congenital muscular dystrophy
      • Congenital myasthenic syndromes
      • Duchenne muscular dystrophy
      • Facioscapulohumeral muscular dystrophy
      • GNE myopathy
      • Limb girdle muscular dystrophy
      • Myotonic dystrophy
      • Myotubular and centronuclear myopathies
      • Spinal muscular atrophy
    • Research Overview
      • DMD
        • Gene Therapy for DMD
        • Mutation Specific Approaches
        • Cell Therapy
        • Drug Therapy
    • Standards of Care & Family Guides
      • CM Care
      • CMD Care
      • DM Care
      • DMD Care
      • BMD Care
      • FSHD Care
      • LGMD Care
      • SMA Care
    • SOP Library
      • MDX Mouse (DMD)
      • GRMD Dog (DMD)
      • CMD Mouse (CMD)
      • SMA Mouse (SMA)
      • CMD animal models
      • MDC1A Preclinical Research
      • Cell Lines
        • Clinical Outcome Measures
        • Functional Evaluation Tools
        • NMR (MRI/MRS) Imaging
        • Muscle Biopsy
    • Social and Ethical Issues
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  • Conference
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