Rare disease
Muscular Dystrophy
Clinical trials recruiting in Australia for Muscular Dystrophy, a rare disease, as the public registries list them.
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At a glance
13 trials recruiting in Australia
186 trials recruiting overseas
Not recorded prevalence
Not recorded age of onset
15 October 2026 date last checked
Trials in Australia
This Australian count is likely to be on the low side. It counts the trials this site files under this condition; the registry’s own search is broader and also returns closely related conditions, some of which have their own page here. If you are searching widely, check the registry as well — every trial below links to its own record.
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Recruiting Not a phased study
Study for people with Muscular Dystrophy
Where- Online Patient Enrollment System, Sydney — Recruiting
Who can join Age child, adult, older adult. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Sanford HealthTimelineStarted 1 July 2010 Today 16 October 2026 Estimated completion 1 December 2100NCT01793168 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Muscular Dystrophy
Where- The Bright Alliance, Randwick (NSW) — Recruiting
Who can join Age 6 Years to 45 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor AMO Pharma LimitedTimelineStarted 23 August 2021 Today 16 October 2026 Estimated completion 31 December 2026NCT05004129 · Checked 11 October 2026
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By invitation Phase 2
Phase 2 study for people with Muscular Dystrophy
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- St Vincent's Hospital, Melbourne (VIC) — this site's status is not stated on the record
Who can join Age child, adult, older adult. Sex Male participants only. Healthy volunteers Not accepted.Sponsor Edgewise Therapeutics, Inc.TimelineStarted 2 November 2023 Today 16 October 2026 Estimated completion 1 February 2031NCT06066580 · Checked 11 October 2026
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By invitation Phase 2
Phase 2 study for people with Muscular Dystrophy
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- Liverpool Hospital, Liverpool (NSW) — this site's status is not stated on the record
- Wesley Research Institute, Auchenflower (QLD) — this site's status is not stated on the record
- Sunshine Coast University Hospital, Birtinya (QLD) — this site's status is not stated on the record
- Alfred Health, Melbourne (VIC) — this site's status is not stated on the record
Who can join Age 16 Years to 70 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Sarepta Therapeutics, Inc.TimelineStarted 19 June 2024 Today 16 October 2026 Estimated completion 31 December 2028NCT06131983 · Checked 12 October 2026
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By invitation Phase 2
Phase 2 study for people with Muscular Dystrophy
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- Liverpool Hospital, Liverpool (NSW) — this site's status is not stated on the record
- Sunshine Coast University Hospital, Birtinya (QLD) — this site's status is not stated on the record
- Royal Brisbane And Women's Hospital, Herston (QLD) — this site's status is not stated on the record
- Alfred Health, Melbourne (VIC) — this site's status is not stated on the record
- Nucleus Network Brisbane Clinic, Herston — this site's status is not stated on the record
- Mater Hospital Brisbane, South Brisbane — this site's status is not stated on the record
Who can join Age 18 Years to 65 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Sarepta Therapeutics, Inc.TimelineStarted 4 March 2024 Today 16 October 2026 Estimated completion 31 December 2026NCT06138743 · Checked 12 October 2026
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Recruiting Phase 2
Phase 2 study for people with Muscular Dystrophy
Where- Investigational Site Number : 0360001, Brisbane (QLD) — Recruiting
Who can join Age 10 Years to 55 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor SanofiTimelineStarted 23 July 2025 Today 16 October 2026 Estimated completion 28 February 2029NCT06844214 · Checked 11 October 2026
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Recruiting Not a phased study
Study for people with Muscular Dystrophy
Where- Murdoch Children's Research Institute, Melbourne — Recruiting
Who can join Age 5 Years to 17 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor University of Kansas Medical CenterTimelineStarted 22 May 2025 Today 16 October 2026 Estimated completion 1 May 2027NCT06847282 · Checked 11 October 2026
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By invitation Phase 2
Phase 2 study for people with Muscular Dystrophy
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- The Royal Children's Hospital, Melbourne (VIC) — this site's status is not stated on the record
- St. Vincent Hospital, Melbourne (VIC) — this site's status is not stated on the record
Who can join Age 16 Years to 40 Years. Sex Male participants only. Healthy volunteers Not accepted.Sponsor Satellos Bioscience, Inc.TimelineStarted 20 August 2025 Today 16 October 2026 Estimated completion 30 September 2027NCT06867107 · Checked 15 October 2026
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By invitation Phase 2
Phase 2 study for people with Muscular Dystrophy
WhereNo Australian site on this record is listed as recruiting right now, even though the trial itself is open. Check with the site or the official record before travelling.
- Wesley Research Institute, Auchenflower — this site's status is not stated on the record
- Neuroscience Clinical Trials Unit, Alfred Brain, Melbourne — this site's status is not stated on the record
Who can join Age 18 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Vertex Pharmaceuticals IncorporatedTimelineStarted 28 April 2025 Today 16 October 2026 Estimated completion 30 January 2029NCT06926621 · Checked 15 October 2026
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Recruiting Phase 3
Phase 3 study for people with Muscular Dystrophy
Where- The Children's Hospital of Westmead, Sydney (NSW) — Recruiting
Who can join Age 7 Years to 11 Years. Sex Male participants only. Healthy volunteers Not accepted.Sponsor Solid Biosciences Inc.TimelineStarted 22 October 2025 Today 16 October 2026 Estimated completion 1 January 2029NCT07160634 · Checked 11 October 2026
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Recruiting Phase 3
Phase 3 study for people with Muscular Dystrophy
Where- St Vincent's Hospital, Melbourne, Fitzroy (VIC) — Recruiting
Who can join Age 16 Years and over. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Dyne TherapeuticsTimelineStarted 14 May 2026 Today 16 October 2026 Estimated completion 1 July 2028NCT07486934 · Checked 15 October 2026
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Recruiting Phase 3
Phase 3 study for people with Muscular Dystrophy
Where- The Children's Hospital at Westmead, Westmead (NSW) — Recruiting
Who can join Age 7 Years to 16 Years. Sex Male participants only. Healthy volunteers Not accepted.Sponsor Avidity Biosciences, Inc.TimelineStarted 1 September 2026 Today 16 October 2026 Estimated completion 1 May 2029NCT07587242 · Checked 15 October 2026
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Recruiting Phase 2
Phase 2 study for people with Muscular Dystrophy
Where- Concord Repatriation General Hospital, Concord (NSW) — Not yet recruiting
- Liverpool Hospital, Liverpool (NSW) — Not yet recruiting
- Wesley Research Institute, Brisbane (QLD) — Not yet recruiting
- Mater Hospital Brisbane, South Brisbane (QLD) — Recruiting
- The Alfred Hospital, Melbourne (VIC) — Recruiting
Who can join Age 18 Years to 65 Years. Sex Anyone, regardless of sex. Healthy volunteers Not accepted.Sponsor Soufflé Therapeutics, Inc.TimelineStarted 1 September 2026 Today 16 October 2026 Estimated completion 1 March 2028NCT07798609 · Checked 15 October 2026
Trials overseas
186 trials for this disease are open to new participants outside Australia, as ClinicalTrials.gov listed them on 11 October 2026. Voxsanity does not publish a page for any of them; the searches below reach them at the registry.
These overseas counts are likely too high. They come from the registry’s own search, which expands a condition name to related conditions, so some of the trials counted are for a different disease in the same family.
Search ClinicalTrials.gov Search the WHO registry portal Joining a trial overseas
Trials not shown here
Some Australian trials are registered only on the Australian New Zealand Clinical Trials Registry and are not shown here. Search them at Australian Clinical Trials.
Key facts
Orphadata Science: Free access data from Orphanet. © INSERM 1999. Available on https://www.orphadata.com. Used under CC BY 4.0. Source: Orphanet / Orphadata (orphadata.com).
About Muscular Dystrophy
The text below is reproduced exactly as its source published it. It has not been edited, shortened or rewritten.
What is muscular dystrophy (MD)? Muscular dystrophy (MD) is a group of more than 30 genetic diseases. They cause weakness of the muscles. Over time, the weakness gets worse and can cause trouble walking and doing daily activities. Some types of MD can also affect other organs. What are the types of muscular dystrophy (MD)? There are many different types of MD. Some of the more common types include: - Duchenne muscular dystrophy, which is the most common childhood form. It is severe and affects boys more often than girls. The symptoms usually start between ages 3 and 6. - Becker muscular dystrophy, which is similar to Duchenne but is less severe and gets worse more slowly. It often starts in the teenage years. - Congenital muscular dystrophies, which are present at birth or before age 2. They can be mild or severe. - Facioscapulohumeral muscular dystrophy, which often starts in the teenage years. At first, it affects the muscles of the face, shoulders, and upper arms. Each of the types of MD can be different in many ways, such as: - Who is more likely to get them - Which muscles they affect - When they appear, such as in infancy, childhood, middle age, or later - What the symptoms are - How serious the symptoms are - How quickly they get worse - Whether they run in families - Whether they affect other organs Even within the same type of MD, people can have different symptoms. What causes muscular dystrophy (MD)? MD is genetic, meaning that it caused by a change in one or more genes. Gene changes are also called gene variants or mutations. The gene changes in MD affect proteins that strengthen and protect muscles. There are different gene changes that cause each type of MD. And sometimes people who have the same type of MD can have different gene changes. Muscular dystrophy can run in families, or you can be the first in your family to have a muscular dystrophy. How is muscular dystrophy (MD) diagnosed? To find out if you or your child has MD, your health care provider may use: - A medical and family history - A physical exam - Blood and urine tests, including genetic tests and tests for certain enzymes that may be released by damaged muscles - Muscle biopsies - Electromyography and nerve conduction studies to find out if muscles are responding the right way to nerve signals - Heart testing, such as an electrocardiogram (EKG), since some types of MD can cause heart problems - Exercise tests to measure muscle strength and breathing and detect any increased rates of certain chemicals following exercise - Imaging tests such as an MRI to look at muscle quality and bulk and measure fatty replacement of muscle tissue What are the treatments for muscular dystrophy (MD)? There is no cure for muscular dystrophy. Treatment can help with the symptoms and prevent complications. It usually includes a combination of therapies, such as: - Physical therapy to help keep muscles flexible and strong - Occupational therapy to relearn lost motor skills and learn ways to work around weakened muscles - Respiratory care, such as breathing exercises, oxygen therapy, and ventilators - Speech therapy to help with speech and swallowing problems - Assistive devices, such as wheelchairs, splints and braces, and walkers - Medicines to help delay damage to muscles or minimize the symptoms of MD - Surgery to treat some of the conditions associated with MD, such as heart problems, scoliosis, and cataracts NIH: National Institute of Neurological Disorders and Stroke
US source; availability in Australia may differ.
Source: MedlinePlus Health Topics (medlineplus.gov), public domain (U.S. National Library of Medicine). Read the full source.
Sources and dates
- ClinicalTrials.gov – trial records, statuses and site lists
- MedlinePlus – the About text above, reproduced exactly
- Orphanet / Orphadata – prevalence, age of onset, inheritance, genes and alternative names, used under CC BY 4.0
- ClinicalTrials.gov processing date: 14 October 2026.
- This page last checked by Voxsanity: 15 October 2026.
Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.
Not medical advice.
Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Orphanet citation
Orphadata Science: Free access data from Orphanet. © INSERM 1999. Available on https://www.orphadata.com. Used under CC BY 4.0. Source: Orphanet / Orphadata (orphadata.com). Accessed 20 September 2026.
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