What changed for Muscular Dystrophy
A plain English summary of what recently moved in the public data Voxsanity tracks for Muscular Dystrophy. Updated from government registries. Not medical advice.
How to read these changes: every figure on this page is the difference between two snapshots of what Voxsanity had tracked on each date. Voxsanity only began taking daily snapshots in July 2026 and is still ingesting the public registries, so a rise here is mostly our own coverage expanding rather than new research appearing. Read it as our tracked coverage growing, not as a claim about real-world research changing this fast: it overstates how much actually changed.
Comparing the two most recent snapshots, 27 July 2026 and 28 July 2026.
- Trials recruiting worldwide 122 to 119 (-3)
- Trials on record 714 to 711 (-3)
Newly subsidised on the PBS
No PBS-reviewed medicines are tracked for Muscular Dystrophy yet, so new PBS listings cannot be reported for it. This is a gap in what Voxsanity has mapped, not a statement that no medicines are subsidised — check the PBS directly.
Past updates
Tracking began in July 2026. Month on month changes will appear here once a second month of data has been recorded. Until then, the comparison above shows the most recent movement.
Recent trend
Weekly, most recent weeks (fills out to a monthly trend as history grows).
Recent trial results
The 30 most recent of 58 trials tracked for Muscular Dystrophy that have reported results to a public registry.
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
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NCT03179631 · results posted 10 March 2026
According to the results reported on ClinicalTrials.gov, this trial enrolled 360 participants in total — 184 in the ataluren group and 176 in the placebo group. The trial ran in two back-to-back phases, each lasting 72 weeks, for a combined total of about three years. The main thing being measured was how far participants could walk in six minutes (known as the "6-minute walk distance" or 6MWD), which is a standard way of tracking physical function over time in conditions that affect movement. A secondary measure looked at how long it took participants to walk or run 10 metres. The reported d...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT05185622 · results posted 24 October 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05185622) enrolled 54 children and young people across six treatment groups, ranging in size from 6 to 16 participants per group. All 54 participants who started the trial completed it — none dropped out. The trial was measuring a range of unwanted medical events (called adverse events) that occurred during the study, as well as any changes in participants' height over 12 weeks. The reported data shows that, when it came to any adverse event occurring during the study, the numbers across the six groups were 7, 9, 6, 4, 3,...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03692312 · results posted 11 September 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT03692312) looked at a drug called tideglusib in people with congenital myotonic dystrophy type 1 (a genetic muscle condition present from birth). A total of 53 people took part — 27 received tideglusib and 26 received a placebo (a dummy treatment with no active ingredient). Of those, 25 in each group completed the study. The trial measured changes in several rating scales filled out by clinicians and caregivers, as well as a short walking test, to see how symptoms and overall condition changed over time. The reported data...
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NCT04281485 · results posted 8 June 2025
According to the results reported on ClinicalTrials.gov, this trial enrolled 114 boys across two groups: 79 in Cohort 1 and 35 in Cohort 2. The trial was measuring changes over 52 weeks (about one year) in physical function, muscle protein levels, and a blood marker related to muscle. The main thing being tracked was performance on the North Star Ambulatory Assessment (NSAA) — a 17-task test of everyday physical skills scored from 0 (unable to do the task independently) to 34 (full independent function), with higher scores meaning better physical ability. By the end of the study, 69 participan...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04572893 · results posted 21 May 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT04572893) tested an investigational drug called MYK-491 in people with a type of heart condition called dilated cardiomyopathy (DCM) — where the heart's main pumping chamber becomes enlarged and weakened. Participants were grouped based on whether their condition was linked to specific gene changes (in genes called MYH7 or TTN, or other genetic causes) or had no identified genetic cause. The trial ran in two stages, called Part A and Part B. In Part A, 41 people started (12 in the MYH7 group, 14 in the TTN group, 5 in the...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04428476 · results posted 24 February 2025
According to the results reported on ClinicalTrials.gov, this trial looked at a treatment called deramiocel and enrolled 13 participants. The trial was measuring two main things over 12 months: how often participants experienced unwanted health events (called adverse events) while on the treatment, and whether there were any changes in arm and hand function over time. The arm function was measured using a scoring tool called the Performance of the Upper Limb Test, Version 2 (PUL 2.0), which gives a score from 0 to 42, where a higher score means better arm and hand function. Notably, the report...
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NCT02485938 · results posted 9 January 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT02485938) enrolled 25 people in total — 12 in the usual care group and 13 in the CAP-1002 (an experimental cell therapy) group. The trial was looking at a range of safety-related measurements, including whether any serious heart-related events occurred shortly after treatment, how often unwanted medical events were reported, and how certain blood test values changed over 6 and 12 months. The reported data shows that when it came to the main heart-related safety events tracked — such as sudden unexpected death or a major h...
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NCT04060199 · results posted 11 December 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04060199) enrolled 77 participants in total — 38 received viltolarsen and 39 received a placebo (an inactive treatment used for comparison). Most participants completed the trial: 36 in the viltolarsen group and 38 in the placebo group. The trial was measuring a physical ability test called the "Time to Stand" (TTSTAND) — specifically, how quickly a participant could get up from lying flat on the floor to a standing position. This was converted into a speed score (rises per second), and the trial tracked how that score cha...
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT03760029 · results posted 19 September 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT03760029) enrolled 312 participants in total across three groups: 99 children who could walk and were under 6 years old, 177 participants who could walk and were aged 6 or older, and 36 participants who could not walk. The study followed participants for up to 30 months and tracked key physical milestones — specifically, the age at which participants lost the ability to walk, stand, or feed themselves. It also used a standardised 17-item physical function test called the Northstar Ambulatory Assessment (NSAA), which scores...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04632940 · results posted 26 August 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04632940) enrolled 73 participants in total — 37 in the pamrevlumab group and 36 in the placebo group — across a 52-week double-blind period followed by a 52-week open-label extension period. The trial was measuring physical functioning in people with Duchenne muscular dystrophy, a condition that progressively affects muscle strength and movement. The main thing being tracked was a score from a 17-item movement assessment called the North Star Ambulatory Assessment (NSAA), which rates everyday physical activities on a scal...
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT04956289 · results posted 20 August 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04956289) enrolled 20 boys or young men receiving the drug viltolarsen at a dose of 80 mg/kg. Half of the participants (10) were able to walk independently (described as "ambulant") and half (10) were not (described as "non-ambulant"). All 20 participants completed the trial. The trial's primary focus was on tracking unwanted medical events — known as "adverse events" — that occurred during treatment, rather than measuring physical function or other health outcomes. The reported data shows that 19 out of 20 participants e...
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NCT04003974 · results posted 10 July 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04003974) enrolled 80 people in total — 40 who took losmapimod (15 mg twice daily) and 40 who received a placebo (a dummy treatment with no active ingredient). The trial was looking at a condition called facioscapulohumeral muscular dystrophy (FSHD), and its main goal was to measure whether losmapimod changed the activity of a protein called DUX4 in muscle tissue. DUX4 is thought to play a role in FSHD, and its activity was estimated by looking at how certain genes behaved in muscle samples taken before and after the treat...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04371666 · results posted 12 March 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04371666) looked at a medicine called pamrevlumab compared to a placebo (an inactive dummy treatment) in people with a muscle-wasting condition. A total of 49 people were assigned to the pamrevlumab group and 49 to the placebo group for the main 52-week treatment period. The trial measured changes in upper limb function, breathing capacity, grip strength, and heart pumping function over that year. The reported data shows that for the primary measure — upper limb function, scored on a 42-point scale where higher scores mea...
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NCT02836418 · results posted 22 December 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT02836418) enrolled 8 participants, all of whom received at least one dose of the study drug, ATYR1940, and all 8 completed the trial. The study was primarily measuring safety-related outcomes — that is, it was tracking whether participants experienced any unwanted medical events, abnormal test results, or other changes while taking the study drug, rather than measuring whether the drug treated a condition. The reported data shows that out of the 8 participants, 7 experienced what are called "treatment-emergent adverse eve...
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NCT03777319 · results posted 23 October 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT03777319) enrolled just two participants — one child with Duchenne muscular dystrophy (DMD) who received spironolactone, and one who received prednisolone (a steroid). Both participants completed the trial. The trial was designed to compare these two medicines in boys with DMD who had not previously taken steroids, by measuring muscle-related outcomes over time. The reported data shows that for the main physical test — how long it took each participant to walk 100 metres — the child on spironolactone improved by 0.6 secon...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03167255 · results posted 18 November 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03167255) enrolled 16 boys with Duchenne muscular dystrophy — 8 in a lower-dose group (40 mg/kg) and 8 in a higher-dose group (80 mg/kg) of the investigational drug NS-065/NCNP-01. All 16 participants completed the trial. The trial was measuring changes in physical movement tasks — specifically how long it took participants to stand up from the floor, run or walk 10 metres, and climb 4 stairs — and comparing those changes to a matched group of historical control participants from an earlier related trial. The trial also re...
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NCT02439216 · results posted 23 September 2022
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called CAT-1004 across three separate stages (called Part A, Part B, and Part C). Part A was a short 7-day open-label phase (meaning everyone knew what they were receiving) involving 17 participants across three dose levels. Part B was a 12-week double-blind phase (where neither participants nor researchers knew who received the medicine or a dummy treatment called a placebo) involving 31 participants. Part C was a longer open-label phase running up to 138 weeks, involving 31 participants — though only 13 of...
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NCT01603407 · results posted 12 August 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT01603407) looked at three different steroid treatment approaches for boys with Duchenne Muscular Dystrophy (DMD): daily prednisone, daily deflazacort, and intermittent (not every day) prednisone. A total of 196 participants were enrolled across the three groups — 65, 65, and 66 respectively — and by the end of the study, 54, 54, and 56 had completed it. The trial measured lung function, movement ability, how satisfied participants were with their treatment, and several other physical measures over up to 36 months. The rep...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03439670 · results posted 13 July 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03439670) enrolled a total of 121 participants across six treatment groups, with group sizes ranging from 15 to 30 people. The trial was measuring how a drug called vamorolone compared to a placebo (an inactive treatment), focusing on a physical ability test called the "Time to Stand" test. This test measures how quickly a person can rise from the floor, recorded in "rises per second" — essentially, a higher number means rising more quickly. The reported data shows that the primary outcome compared changes in the Time to...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03783923 · results posted 27 June 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03783923) enrolled 11 participants in total — 5 in the deflazacort group and 6 in the placebo group — for the first 26-week placebo-controlled phase. The trial was measuring how a medicine called deflazacort compared to a dummy treatment (placebo) across a range of physical movement tasks, including climbing and descending stairs, walking, and getting up and moving around. The number of people who completed the trial was very small: only 1 in the deflazacort group and 2 in the placebo group finished the first phase, and no...
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NCT03703882 · results posted 21 June 2022
According to the results reported on ClinicalTrials.gov, this trial enrolled 131 boys with Duchenne muscular dystrophy (DMD) — 88 in the treatment group (Dose 1) and 43 receiving a placebo (an inactive dummy treatment). By the end of the study, 85 in the treatment group and 37 in the placebo group completed it. The trial ran for 52 weeks and was measuring physical function — specifically, whether the boys' ability to move and perform everyday physical tasks changed over time. The main tool used was something called the North Star Ambulatory Assessment (NSAA), a 17-item checklist where a traine...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03796637 · results posted 5 April 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03796637) enrolled 6 participants, all of whom completed the study. All 6 were in a single group receiving a medicine called ataluren. The trial was measuring levels of a protein called dystrophin in muscle tissue, using two different laboratory methods. Dystrophin is a protein found in muscles, and the trial tracked how much of it could be detected after treatment. The reported data shows that dystrophin levels were measured in two specific muscles — the gastrocnemius (calf muscle) and the tibialis anterior (a muscle in...
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NCT01976091 · results posted 1 April 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT01976091) involved six participants in total, divided into three small groups testing a treatment called SRP-9004. One person was in Cohort 1A, three were in Cohort 1B, and two were in Cohort 2. All six participants received at least one dose of the study drug, and all six completed the trial. The trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) after receiving the treatment. A secondary measure looked at whether participants' ability to walk changed, using a s...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03531788 · results posted 18 February 2022
According to the results reported on ClinicalTrials.gov, this trial looked at two types of arm support devices — the Armon Ayura (made by Kinova) and the JAECO WREX — designed to help people move their arms more easily. A total of 18 people were enrolled: 12 in the Armon Ayura group and 6 in the JAECO WREX group. Of those, 17 completed the trial (11 in the Armon Ayura group and all 6 in the JAECO WREX group). The trial measured arm movement using a wrist-worn activity tracker, and also asked participants to set personal goals and rate how well those goals were met with and without the device....
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NCT00577577 · results posted 6 January 2022
According to the results reported on ClinicalTrials.gov, this trial enrolled 69 people in total — 34 in the IPLEX™ group and 35 in the placebo group. The trial was measuring physical activity and movement in people over a 24-week period. The main things being measured included how far participants could walk in six minutes, how many steps they took each day, how quickly they walked during their busiest periods of the day, how much time they spent inactive, and how fast they could climb up and down four stairs. Of the 69 who started, 59 completed the trial (29 in the IPLEX™ group and 30 in the...
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NCT02606136 · results posted 30 December 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT02606136) enrolled 21 people who all received at least one dose of the study drug, pamrevlumab. The trial ran for up to 104 weeks (about two years), with an optional extension period of up to 208 weeks. Of the 21 who started the main study, 15 completed it and then entered the extension phase; however, none completed the extension phase. The trial was measuring changes over time in lung function, heart function, and upper body strength and movement in participants. The reported data shows that, on average, participants' l...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01856868 · results posted 22 December 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT01856868) involved 7 participants, all of whom received a treatment called epicatechin — a natural compound found in foods like dark chocolate and green tea. All 7 participants completed the trial, and none dropped out. The trial was measuring changes in certain proteins found in muscle tissue over 8 weeks. These proteins are involved in processes like how muscles produce energy and how muscle growth is regulated. The reported data shows the levels of six different muscle proteins, measured before and after the 8-week tre...
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NCT02239224 · results posted 11 August 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 20 people in total across four groups. Three participants received a low dose of the study drug ATYR1940 (0.3 mg/kg), six received a medium dose (1.0 mg/kg), six received a higher dose (3.0 mg/kg), and five received a placebo (an inactive treatment given for comparison). The trial was primarily measuring a range of safety-related observations — including any unwanted medical events that occurred after dosing, blood and urine test results, physical examination findings, vital signs such as heart rate and blood pressure...
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NCT02740972 · results posted 12 July 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT02740972) looked at a drug called NS-065/NCNP-01, which was being tested in people with Duchenne muscular dystrophy (DMD) — a genetic condition that affects muscle. The trial ran in two parts: a four-week blinded phase (where some participants received a placebo and others received one of two doses of the drug — 40 mg/kg or 80 mg/kg), followed by a 20-week open-label phase (where all participants received one of the two doses and everyone knew what was being given). A total of 16 participants took part across both phases....
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04054375 · results posted 8 July 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 20 participants, all of whom completed the study with no drop-outs. The trial involved a single group who received a weekly steroid treatment. It was measuring several health markers in people with dysferlinopathy (a rare inherited muscle condition), including blood sugar levels, a longer-term blood sugar measure (HbA1c), cholesterol levels, a muscle enzyme called creatine kinase (a substance released into the blood when muscle is damaged), lung function, and physical ability scores. Two sets of measurements were reco...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
See all 58 reported results for Muscular Dystrophy
These are the results as reported to the registry, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
What is coming
AI generated pipeline summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to NIH funding data, Muscular Dystrophy receives a very large amount of public research funding in the United States, and the level of that investment has remained broadly steady year on year. For patients, this signals that the condition is taken seriously by major public funders and that scientific attention to it has not dropped away. A stable and substantial funding base generally su...
See the full Muscular Dystrophy page
Not medical advice. Voxsanity republishes public government health data in plain English. These figures describe research and registry activity, not the suitability of any treatment for any person. Trial status can change; always verify current status directly with the trial site and confirm Australian availability with your doctor.