New hope for duchenne: experimental drug targets genetic glitch
NCT ID NCT05996003
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an experimental drug called NS-089/NCNP-02 (Brogidirsen) in 20 boys with Duchenne muscular dystrophy whose genetic mutation can be fixed by skipping exon 44. The drug is given as a weekly IV infusion and aims to help the body produce a working version of the dystrophin protein. Researchers will check safety, how the drug moves through the body, and whether it increases dystrophin levels in muscle.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NS-089/NCNP-02 (Brogidirsen)
What this could lead to
If successful, this could lead to a treatment that helps boys with Duchenne muscular dystrophy produce a shorter but functional dystrophin protein, potentially slowing muscle decline.
What could go wrong
This is an early Phase 2 trial with only 20 participants, so results may not apply to all. The drug may not produce enough dystrophin to meaningfully change the disease course, and side effects are still being studied.
Disclaimer
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This is a summary of
the original study
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Alberta Children's Hospital
NOT_YET_RECRUITINGCalgary, Alberta, Canada
Contact Email: •••••@•••••
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Ankara Bilkent City Hospital
NOT_YET_RECRUITINGAnkara, 06800, Turkey (Türkiye)
Contact Email: •••••@•••••
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Ann and Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Email: •••••@•••••
Contact Email: •••••@•••••
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British Columbia Children's Hospital
NOT_YET_RECRUITINGVancouver, British Columbia, Canada
Contact Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Email: •••••@•••••
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Fukui Prefectural Hospital
NOT_YET_RECRUITINGFukui-shi, Fukui, 910-8526, Japan
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London Health Sciences Centre
RECRUITINGLondon, Ontario, Canada
Contact Email: •••••@•••••
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NHO Osaka Toneyama Medical Center
NOT_YET_RECRUITINGToyonaka, Osaka, 560-8552, Japan
Contact Email: •••••@•••••
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National Center of Neurology and Psychiatry
NOT_YET_RECRUITINGKodaira, Tokyo, 187-8551, Japan
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National Hospital Organization Nagara Medical Center
RECRUITINGNagara, Gifu-shi, Gifu, 502-8558, Japan
Contact Email: •••••@•••••
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Perth Children's Hospital
NOT_YET_RECRUITINGNedlands, Western Australia, 6009, Australia
Contact Email: •••••@•••••
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Pusan National University Yangsan Hospital
NOT_YET_RECRUITINGYangsan, Gyeongsangnam, South Korea
Contact Email: •••••@•••••
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Email: •••••@•••••
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Rare Disease Research, LLC - FL
RECRUITINGKissimmee, Florida, 34746, United States
Contact Email: •••••@•••••
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S.B.U. Dr. Behcet uz Pediatric Diseases and Surgery Training and Research Hospital
NOT_YET_RECRUITINGIzmir, 11794, Turkey (Türkiye)
Contact Email: •••••@•••••
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Seoul National University Hospital
NOT_YET_RECRUITINGSeoul, South Korea
Contact Email: •••••@•••••
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Shiga General Hospital
NOT_YET_RECRUITINGMoriyama-shi, Shiga, 524-8524, Japan
Contact Email: •••••@•••••
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Starship Children's Hospital
NOT_YET_RECRUITINGAuckland, 1023, New Zealand
Contact Email: •••••@•••••
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The Children's Hospital of Philadelphia (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Email: •••••@•••••
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UT Southwestern/Children's Health
RECRUITINGDallas, Texas, 75207, United States
Contact Email: •••••@•••••
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University of Kansas Medical Center (KUMC)
RECRUITINGKansas City, Kansas, 66160, United States
Contact Email: •••••@•••••
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University of Pittsburgh School of Medicine
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Email: •••••@•••••
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Virginia Commonwealth University Health System
RECRUITINGRichmond, Virginia, 23298, United States
Contact Email: •••••@•••••
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Yeditepe University Kosuyolu Hospital
NOT_YET_RECRUITINGIstanbul, 34718, Turkey (Türkiye)
Contact Email: •••••@•••••