New hope for duchenne: targeted therapy ENTR-601-45 enters human trials
NCT ID NCT07038824
First seen Jan 07, 2026 · Last updated May 23, 2026 · Updated 25 times
Summary
This study tests an experimental drug called ENTR-601-45 in 24 people with Duchenne muscular dystrophy who have a specific genetic mutation (exon 45 skipping). The goal is to check the drug's safety, find the right dose, and see if it can help muscles work better. Participants receive the drug or a placebo through an IV, and researchers compare muscle biopsies and physical tests over time.
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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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Contacts and locations
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Locations
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Alder Hey Children's NHS Foundation Trust
NOT_YET_RECRUITINGLiverpool, L122AP, United Kingdom
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Centre Hospitalier Régional de la Citadelle
RECRUITINGLiège, 4000, Belgium
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Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
RECRUITINGRome, 00168, Italy
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Great Ormond Street Hospital for Children
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
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Hospital Universitario Vall d'Hebron
RECRUITINGBarcelona, 08035, Spain
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IRCCS Ospedale San Raffaele
RECRUITINGMilan, 20132, Italy
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Leeds General Infirmary
RECRUITINGLeeds, LS1 3EX, United Kingdom
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Leids Universitair Medisch Centrum
RECRUITINGLeiden, 2333 ZA, Netherlands
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Ospedale Pediatrico Bambino Gesu
RECRUITINGRome, 00165, Italy
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Oxford University Hospitals NHS Foundation Trust
RECRUITINGOxford, OX3 9DU, United Kingdom
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Royal Manchester Children's Hospital
NOT_YET_RECRUITINGManchester, M13 9WL, United Kingdom
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Stichting Radboud Universitair Medisch Centrum
RECRUITINGNijmegen, 6525 GA, Netherlands
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UZ Leuven
RECRUITINGLeuven, 3000, Belgium
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University Hospital Gent
RECRUITINGGhent, 9000, Belgium
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Conditions
Explore the condition pages connected to this study.