ArticleBrain and behavior2025
Long-Term Effects of Nusinersen Dosing Frequency on Adult Patients With Spinal Muscular Atrophy: Efficacy of a 6-Month Dosing Interval.
Article in Brain and behavior, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed.
- Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances.Therapeutic advances in neurological disorders · 2026Review
- Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations.Frontiers in pharmacology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
Abstract
objectiveSpinal muscular atrophy (SMA) is a genetic disease caused by the degeneration of spinal motor neurons due to a deficiency in survival motor neuron protein (SMN) protein, leading to progressive muscle atrophy and weakness. nusinersen, an antisense oligonucleotide that increases SMN protein expression, has shown effectiveness in both pediatric and adult patients with SMA. While it is administrated every 4 months during the maintenance period in most countries, the dosing interval is 6 months in Japan. The impact of this dosing difference on long-term outcomes is not fully understood. This study evaluates the long-term efficacy of the 6-month dosing protocol of nusinersen in adult SMA patients.
methodsWe assessed 14 adult patients treated with nusinersen every 6 months over a period of up to 39 months using the Hammersmith Function Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM). The results were compared with those from a recent cohort study of adult SMA patients in Europe.
resultsFor ambulatory patients, the mean changes in HFMSE scores at 15, 27, and 39 months were 6.7, 8.3, and 8.0 points, respectively. These results were similar to those observed in the European cohort. In contrast, for nonambulatory patients, the mean changes in HFSME scores were -0.3, -1.4, and -1.3 points, and the mean changes in RULM scores were 2.0, 0.5, and 1.0 points at the same time points. These results were generally less favorable compared to the European cohort but did not reach clinically meaningful deterioration. DISCUSSION: The findings of this study suggest that the 6-month nusinersen dosing protocol provides sustained long-term benefits for ambulatory adult SMA patients. For nonambulatory patients, the 6-month protocol appears less effective than the 4-month protocol. We believe that future nusinersen treatment strategies for adult SMA patients should be flexible, with adjustments based on disease severity. In particular, increasing the dosing frequency and/or dosage in nonambulatory patients may lead to greater improvements.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.