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New hope for duchenne: targeted therapy now available for eligible patients

NCT ID NCT07250737

AVAILABLE Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 13, 2026 · Updated 8 times

Summary

This program provides access to an experimental drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. The goal is to help control the disease and improve muscle function. Participants must be at least 2 years old and live in the US. This is not a cure, but aims to manage the condition long-term.

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Conditions

The condition(s) this trial relates to.

atrophic muscular disease Duchenne muscular dystrophy hereditary disease muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder nervous system disorder neuromuscular disease X-linked disease

As listed by the trial registrant

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

GENETIC DISEASES, INBORN GENETIC DISEASES, X-LINKED MUSCULAR DISEASES MUSCULAR DISORDERS, ATROPHIC MUSCULAR DYSTROPHIES MUSCULAR DYSTROPHY, DUCHENNE MUSCULOSKELETAL DISEASES NERVOUS SYSTEM DISEASES NEUROMUSCULAR DISEASES

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Arkansas Children's Hospital

    AVAILABLE

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • Connecticut Children's Medical Center

    AVAILABLE

    Hartford, Connecticut, 06106, United States

  • Gillette Children's

    AVAILABLE

    Saint Paul, Minnesota, 55101, United States

  • Nationwide Children's Hospital

    AVAILABLE

    Columbus, Ohio, 43205, United States

  • Neurology & Neuromuscular Care Center

    AVAILABLE

    Flower Mound, Texas, 75028, United States

  • Nicklaus Children's Hospital

    AVAILABLE

    Miami, Florida, 33155, United States

  • Prisma Health-Midlands Children's Hospital

    AVAILABLE

    Columbia, South Carolina, 29203, United States

  • Rare Disease Research

    AVAILABLE

    Kissimmee, Florida, 34746, United States

  • Rare Disease Research

    AVAILABLE

    Atlanta, Georgia, 30329, United States

  • Rare Disease Research

    AVAILABLE

    Iselin, New Jersey, 08830, United States

  • UC San Diego Health

    AVAILABLE

    San Diego, California, 92121, United States

  • UCI Alpha Clinic

    AVAILABLE

    Irvine, California, 92612, United States

  • UMass Memorial Health Care

    AVAILABLE

    Worcester, Massachusetts, 01583, United States

  • University of Alabama

    AVAILABLE

    Birmingham, Alabama, 35294, United States

  • University of Kansas Medical Center

    AVAILABLE

    Kansas City, Kansas, 66103, United States

  • University of Pennsylvania

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

  • University of Vermont Medical Center

    AVAILABLE

    Burlington, Vermont, 05401, United States

  • Washington University

    AVAILABLE

    St Louis, Missouri, 63110, United States

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