New drug hopes to slow duchenne in boys with specific gene mutation
NCT ID NCT07037862
First seen Nov 21, 2025 · Last updated May 20, 2026 · Updated 21 times
Summary
This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). The goal is to see if the drug is safe, tolerable, and can help improve muscle function compared to a placebo. Participants receive the drug through an IV and will have regular checkups, including muscle biopsies, to measure any changes.
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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 Serena Onlus - Centro Clinico NeMO Milano
RECRUITINGMilan, 20162, Italy
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Freeman Hospital
RECRUITINGNewcastle upon Tyne, NE1 3BZ, United Kingdom
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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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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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UZ Leuven
RECRUITINGLeuven, 3000, Belgium
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University Hospital Gent
RECRUITINGGhent, 9000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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