New hope for duchenne: experimental drug targets genetic flaw
NCT ID NCT07037862
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests an investigational drug called ENTR-601-44 in 24 boys and young men with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping a specific part of the gene (exon 44). The trial has two parts: first, to find the safest and most effective dose, and second, to check how well it works. Participants receive multiple IV infusions and are monitored with blood tests, muscle biopsies, and physical exams.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ENTR-601-44 (a drug designed to help muscle cells produce a shortened but functional dystrophin protein)
What this could lead to
If successful, this could lead to a treatment that slows muscle decline and improves function in people with Duchenne muscular dystrophy who are eligible for exon 44 skipping.
What could go wrong
This is an early-phase trial with only 24 participants, so results may not apply to everyone. The drug may cause side effects or fail to show meaningful benefit.
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Long-Term DMD Study of ENTR-601-44/45 (NCT07682129)
- DMD Heart Function Study Using MRI (NCT07674758)
- Digital Monitoring for Upper Limb Function in DM NCT07664124
- GLP-1 Drug Safety in Duchenne Muscular Dystrophy NCT07642635
- DYNE-251 for Duchenne Muscular Dystrophy (NCT07608432)
- Duchenne EHR Study: Better Data for Better Care NCT07609394
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
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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
Contact Phone: •••-•••-•••• Email: •••••@•••••
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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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