What changed for Spinal Muscular Atrophy
A plain English summary of what recently moved in the public data Voxsanity tracks for Spinal Muscular Atrophy. 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 at Australian sites 3 to 4 (+1)
- Trials recruiting worldwide 77 to 79 (+2)
- Trials on record 414 to 417 (+3)
Newly subsidised on the PBS
No medicines were newly subsidised on the PBS for Spinal Muscular Atrophy in July 2026. Voxsanity tracks 3 PBS-subsidised medicines for this condition; none of them was listed for the first time this month.
Source: Pharmaceutical Benefits Scheme (PBS), © Commonwealth of Australia. Data used and redistributed under permission; not modified from its original wording where displayed verbatim.
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 50 trials tracked for Spinal Muscular Atrophy 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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NCT05430113 · results posted 16 March 2026
According to the results reported on ClinicalTrials.gov, this trial looked at the use of spinal cord stimulation (a technique that delivers mild electrical signals to the spine) in a very small group of 3 participants. All 3 people completed the study. The trial was measuring whether turning the stimulation on made any difference to leg muscle strength, movement, walking distance, and fatigue, compared to when the stimulation was turned off. The reported data shows that for the main (primary) measure of muscle strength — specifically the force produced at the hip — 2 out of 3 participants met...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT06411912 · results posted 3 March 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT06411912) enrolled 54 people in total — 26 received a placebo and 28 received the investigational drug NIDO-361. The trial was studying a condition called Spinal and Bulbar Muscular Atrophy (SBMA), a rare inherited muscle-wasting disease. The main things being measured were changes in lean muscle volume across the thighs and whole body (assessed by how much fat had replaced muscle tissue), as well as how many participants experienced unwanted health events (adverse events) during the study. Secondary measurements included...
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NCT05089656 · results posted 8 December 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05089656) enrolled 126 children aged 2 to under 18 years with spinal muscular atrophy (SMA). It was a crossover trial, meaning participants were split into two groups: 75 children received OAV101 (an investigational gene therapy) first for 52 weeks, followed by a sham (inactive) procedure, while 51 children received the sham procedure first, then OAV101. The main thing being measured was change in a motor ability score called the HFMSE — a 66-point scale used specifically in SMA where higher scores mean greater physical ab...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04090528 · results posted 5 December 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT04090528) enrolled 60 people with prostate cancer, split evenly into two groups of 30. One group received a single DNA vaccine (called pTVG-HP) combined with a drug called pembrolizumab, while the other group received two DNA vaccines (pTVG-HP and pTVG-AR) combined with pembrolizumab. The main thing the trial was measuring was how many people in each group had not seen their cancer get worse, and had not died, at the six-month mark — a measure known as "progression-free survival rate." By the end of the study, 21 people in...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02176863 · results posted 5 December 2025
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called Flebogamma® 5% DIF (an intravenous immunoglobulin, a product made from donated blood plasma) against a placebo (an inactive treatment) in people with a particular condition affecting physical function. The trial was run in two stages and used different doses — 2 grams per kilogram of body weight and 1 gram per kilogram. In total, 191 people started the trial across all five groups: 42 in each of the three Stage 1 groups and 33 and 32 in the two Stage 2 groups. The number who completed the trial varied...
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NCT02386553 · results posted 20 October 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT02386553) enrolled 25 infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through genetic testing rather than because they were already unwell. The babies were divided into two groups based on how many copies they had of a gene called SMN2 (15 babies with 2 copies, and 10 with 3 copies), as this gene affects how the condition may develop. The trial was testing a medicine called ISIS 396443 (also known as nusinersen) and was primarily measuring the...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03032172 · results posted 1 October 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT03032172) enrolled a total of 174 participants across four treatment groups during the main treatment period: 13 received RO6885247, 76 received nusinersen, 71 received olesoxime, and 14 received AVXS-101. The trial was an observational study tracking participants who were already receiving one of these four medicines for spinal muscular atrophy (SMA). After the main treatment period, those who completed it could continue into an open-label extension phase — meaning everyone knew which medicine was being taken — with 154 p...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04089566 · results posted 5 June 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT04089566) looked at nusinersen, a medicine used for spinal muscular atrophy (SMA) — a condition that affects muscle strength and movement. The trial was run in three parts (A, B, and C) and tested different dose levels of the medicine. In total, 145 people took part across all groups. Part A included 6 people, Part B included 99 people (split between those with an early-onset and later-onset form of SMA), and Part C included 40 people. Not everyone who started the trial completed it — for example, in one of the Part B infa...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT05386680 · results posted 3 June 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05386680) enrolled 27 participants who received a single dose of the gene therapy OAV101 (at a dose level of 1.2×10¹⁴ viral genome particles). Of the 27 who started, 25 completed the study and 2 did not. The trial was primarily focused on tracking unintended medical events (called adverse events) that occurred after treatment, and secondarily on measuring changes in participants' physical abilities over 52 weeks using two standard movement assessment tools used in spinal muscular atrophy (SMA). The reported data shows tha...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04926181 · results posted 15 May 2025
According to the results reported on ClinicalTrials.gov, this trial enrolled just 2 participants, both of whom completed the study. The trial was testing a combination of two medicines — apalutamide and cetrelimab — in a single group, with no comparison group. The main thing the trial set out to measure was a "composite response rate" — meaning whether participants showed both a significant drop in a prostate cancer marker in the blood (called PSA) and a shrinkage of tumours visible on scans. The trial also tracked a number of secondary measures, including side effects linked to the treatment,...
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NCT02268552 · results posted 9 April 2025
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called LMI070 in people with a condition the trial was designed to study. The trial ran in two main parts plus an extension phase. In Part 1, small groups of 2–4 participants each received one of five different doses of LMI070 (measured in milligrams per square metre of body surface area), giving a total of 13 participants in that part. In Part 2, 25 participants were divided into two groups receiving different doses measured by body weight (0.625 mg/kg and 2.5 mg/kg). The trial was measuring, among other thi...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02594124 · results posted 22 October 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT02594124) enrolled a total of 292 participants across nine groups. The participants had either infantile-onset or later-onset Spinal Muscular Atrophy (SMA) and came from several earlier related studies. The groups included people who had previously received a placebo-like "sham" procedure, people who had previously received the study drug nusinersen (also referred to as ISIS 396443), and people entering from other studies. The trial was measuring a range of monitored observations — including unwanted medical events (called...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01645787 · results posted 3 September 2024
According to the results reported on ClinicalTrials.gov, this trial enrolled 11 people in total — six in one group and five in the other. It used a "crossover" design, meaning everyone took turns receiving the study drug (4-aminopyridine, also known as Ampyra) and a placebo (a dummy pill with no active ingredient), so each person experienced both treatments at different points. The trial was looking at whether the drug made any difference to walking ability and muscle function in people with Spinal Muscular Atrophy (SMA) Type 3, a condition that affects muscle strength and movement. The study...
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NCT05073133 · results posted 31 May 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT05073133) involved 16 participants who received a treatment called OAV101. The trial was primarily measuring the number of participants who experienced medical events (called adverse events, or AEs) after receiving the treatment — that is, any unwanted sign, symptom, or health change that occurred during the study period. It also tracked a set of specific health concerns the researchers were watching closely, and as a secondary measure, it looked at how many children reached certain movement milestones (such as sitting, cr...
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NCT03779334 · results posted 5 March 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT03779334) looked at a medicine called risdiplam in infants who had been diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms — meaning they were identified through newborn screening. A total of 26 babies took part, split into three groups based on how many copies of a gene called SMN2 they carried: 8 babies had 2 copies, 13 had 3 copies, and 5 had 4 or more copies. The trial's main question was focused specifically on a smaller subgroup of 5 babies from the 2-copy group who met particular criteria a...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04851873 · results posted 5 January 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04851873) enrolled 24 children in total, divided into three groups based on body weight: 7 children weighing 8.5–13 kg, 8 children weighing more than 13–17 kg, and 9 children weighing more than 17–21 kg. All 24 children completed the study. Each child received a single dose of OAV101 (a gene therapy), and the trial was primarily measuring how many participants experienced unwanted medical events (called adverse events) and changes in vital signs such as blood pressure and breathing rate across the different weight groups....
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03819660 · results posted 30 November 2023
According to the results reported on ClinicalTrials.gov, this trial enrolled 13 participants, all of whom completed the study with no drop-outs. The trial was looking at a medicine called amifampridine phosphate in people with Spinal Muscular Atrophy (SMA) Type 3 — a condition that affects muscle strength and movement. The study was primarily tracking how many participants experienced unwanted side effects (called treatment-emergent adverse events) over the long term, and it also measured changes in participants' quality of life using a standard questionnaire. The reported data shows that 5 o...
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NCT03653390 · results posted 21 November 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT03653390) involved 505 people in total across three groups: 183 people in the "EnhanceWellness for Disability" (EW-D) programme, 159 in a "Wellness Education" group, and 163 in a control group (who received neither programme). The trial was measuring whether taking part in these programmes made a difference to things like people's ability to join in community and social activities, their confidence in managing a long-term health condition, how much pain and fatigue got in the way of daily life, their psychological resilien...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03381729 · results posted 16 February 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT03381729) enrolled 32 children with spinal muscular atrophy (SMA) across several groups, divided by age and by the dose of the gene therapy AVXS-101 (also known as onasemnogene abeparvovec) they received. The younger children were aged 6 months to under 2 years, and the older children were aged 2 years to under 5 years. All 32 children who started the trial completed it. The trial was measuring things like the ability to stand or walk independently, changes in a motor function score, how many children experienced medical e...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03921528 · results posted 17 November 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03921528) looked at a treatment for Spinal Muscular Atrophy (SMA) across three groups of participants, divided by their type of SMA and mobility level. In total, 58 people started the trial: 11 in Cohort 1 receiving the study drug alone, 12 in Cohort 1 receiving it alongside another SMA treatment, 15 in Cohort 2, and 10 each in two Cohort 3 groups (a lower dose and a higher dose). The trial measured changes in physical ability scores over 12 months using standardised movement assessments — tools that assign a number to how...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03986671 · results posted 21 June 2022
According to the results reported on ClinicalTrials.gov, this trial enrolled a total of 50 participants across three groups: 36 people described as normal (healthy) subjects, 9 people with Amyotrophic Lateral Sclerosis (ALS, a neurological condition affecting muscle control), and 5 people with Obstructive Sleep Apnea (a condition where breathing repeatedly stops during sleep). The trial was measuring whether a sensor-based testing method called TM-EMG — a way of recording muscle activity in the throat and tongue — produced consistent results compared to an existing standard method (NEMG, anoth...
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NCT03837184 · results posted 11 January 2022
According to the results reported on ClinicalTrials.gov, this trial involved just 2 participants, both of whom completed the study. The trial was testing a treatment called AVXS-101 and was measuring two things: whether participants could sit up on their own for at least 10 seconds without support, and whether participants reached 14 months of age without dying, needing permanent breathing support, or withdrawing from the study. The reported data shows that 1 out of the 2 participants was able to sit upright without support for at least 10 seconds — this was confirmed by video recording using...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03505099 · results posted 11 January 2022
According to the results reported on ClinicalTrials.gov, this trial (NCT03505099) involved 29 babies in total, split into two groups based on their genetic profile. Fourteen babies were in Cohort 1 (carrying two copies of a gene called SMN2) and fifteen were in Cohort 2 (carrying three copies of SMN2). All 29 participants completed the study. The trial was measuring whether babies reached certain physical milestones — such as sitting, standing, and walking — that are not typically expected in children with their condition. The reported data shows that for Cohort 1, all 14 participants were re...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT03461289 · results posted 2 April 2021
According to the results reported on ClinicalTrials.gov, this trial (NCT03461289) enrolled 33 children who received a treatment called onasemnogene abeparvovec-xioi. The trial was looking at two main things: first, how many children could sit up on their own for at least 10 seconds without using their arms or hands to balance (the primary, or main, goal); and second, how many children were still alive, not needing a permanent breathing machine, and still in the study by the time they reached 14 months of age. Thirty-two of the 33 children completed the study, and one did not. The reported dat...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01422200 · results posted 19 October 2020
According to the results reported on ClinicalTrials.gov, this trial enrolled 22 participants in total — 11 in a group that received a flu vaccine by subcutaneous injection (under the skin) and 11 in a group that received the same vaccine by intramuscular injection (into the muscle). All 22 participants completed the study. The trial was measuring how the body's immune response — specifically the level of flu-fighting antibodies in the blood — compared between the two injection methods, across three different flu strains included in the vaccine. The reported data shows antibody responses using...
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NCT03988907 · results posted 19 October 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT03988907) involved a total of 35 participants split into two parts — 8 people in Part 1 and 27 people in Part 2. All 8 participants in Part 1 completed the study, as did 26 of the 27 in Part 2 (one person did not complete Part 2). The trial was measuring how a drug called risdiplam — used for spinal muscular atrophy — affects the way the body processes another drug called midazolam when both are taken together. Specifically, researchers tracked how much midazolam and one of its breakdown products (called 1-hydroxy midazola...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02644668 · results posted 31 August 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT02644668) looked at a medicine called reldesemtiv in people with spinal muscular atrophy (SMA), a condition that affects muscle strength and movement. A total of 70 people took part — 26 received a placebo (a dummy treatment with no active ingredient), 24 received reldesemtiv at a lower dose (150 mg twice daily), and 20 received reldesemtiv at a higher dose (450 mg twice daily). The trial ran for 8 weeks and measured several things related to breathing and muscle function, including how much air participants could breathe...
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NCT03306277 · results posted 16 July 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT03306277) enrolled 22 infants who received a single treatment called onasemnogene abeparvovec-xioi (a gene therapy). The trial was studying children with a serious muscle-weakening condition called spinal muscular atrophy (SMA). The study had two main things it was measuring: whether the children could sit up on their own for at least 30 seconds, and whether they survived without needing permanent breathing support. Nineteen of the 22 participants completed the study, and three did not. The reported data shows that 13 out...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00568802 · results posted 2 October 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT00568802) enrolled 27 participants in total, all grouped together under a single study arm for tracking purposes. Twenty-four participants completed the study, while three did not finish. The trial was comparing hydroxyurea (a medicine) against a placebo (a dummy treatment with no active ingredient) in participants with spinal muscular atrophy, a condition affecting muscle strength and movement. The study set out to measure two main things: how well participants could perform motor tasks (such as movement tests and timed p...
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NCT00568698 · results posted 2 October 2019
According to the results reported on ClinicalTrials.gov, this trial (NCT00568698) enrolled 29 participants in total. It was studying hydroxyurea compared to a placebo (an inactive dummy treatment) in what appears to have been a trial involving children with spinal muscular atrophy, a condition affecting muscle strength and movement. The trial aimed to measure both how safe the treatment appeared to be and whether it had any effect on survival and breathing function, as well as certain biological markers in the body. Of the 29 people who started the trial, 18 completed it and 11 did not. The r...
See all 50 reported results for Spinal Muscular Atrophy
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, a substantial level of public research investment is currently directed toward Spinal Muscular Atrophy, and encouragingly, that funding has been rising year on year. This upward trend suggests that the scientific and medical community continues to see meaningful value in pursuing new understanding of this condition. For patients, rising public investment generally me...
See the full Spinal Muscular Atrophy 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.