Spinal muscular atrophy therapies
Gene therapy and other disease-modifying treatments for SMA.
Papers per year
Each bar is 12 months; the last one ends 17 Oct 2026. New trials in the same years: 16, 18, 12, 6, 16.
All papers from the last 12 months
247 papers (18 Oct 2025 – 17 Oct 2026), newest first. Loading the full list…
- Respiration during sleep in children with spinal muscular atrophy type 2 and 3, treated with nusinersen or risdiplamSleep Med2026 DecPMID 42710465
- Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatmentRNA Biol2026 DecPMID 42149739Review
- UK SMA patient registry: a 3-year patient-reported outcome measures study supporting the drug appraisal of Nusinersen and Risdiplam in spinal muscular atrophyJ Neurol Sci2026 Nov 15PMID 42727545
- Compound muscle action potential amplitudes in newborn screen positive spinal muscular atrophyClin Neurophysiol2026 NovPMID 42541962
- Spinal Muscular Atrophy in Adult Neurology Services in IndiaClin Genet2026 NovPMID 42536319
- Sleep disordered breathing and autonomic outcomes in adult patients with spinal muscular atrophy (SMA) receiving RisdiplamSleep Med2026 NovPMID 42526101
- Consensus on the use of intrathecal gene therapy for spinal muscular atrophyNeuropediatrics2026 Oct 13PMID 42869088
- Astrocyte SMN Restoration and Forskolin Treatment Improve SMA Synaptic PathologyAnn Neurol2026 Oct 12PMID 42861773
- Long-term real-world outcomes of nusinersen treatment in patients with spinal muscular atrophy types I-IIIBrain Dev2026 Oct 5PMID 42833105
- Pathophysiology and biomaterials based therapeutic strategies for spinal muscular atrophyIn Vitro Model2026 OctPMID 42763486Review
More pediatrics trends
All specialties →Kangaroo mother care
Skin-to-skin contact for preterm and low birth weight infants.
RSV prevention in infants
Nirsevimab, maternal vaccination and other ways to protect infants from RSV.
Key point: A complementary, progressive strategy should integrate maternal vaccination and monoclonal antibody immunoprophylaxis, initially prioritizing high-risk infants and gradually expanding to all susceptible infants.
Food allergy immunotherapy
Oral immunotherapy and biologics for food allergy.
Social media and youth mental health
Screen time and social media use and their links to anxiety and depression in young people.
Neonatal therapeutic hypothermia
Cooling newborns after birth asphyxia to protect the brain.
MIS-C
Multisystem inflammatory syndrome in children after SARS-CoV-2.
Too many papers to read?
PubDaily picks the ones that matter in your specialty and summarises them, about 10 minutes a week.
How these numbers are counted
PubMed query, searched in titles and abstracts by publication date:
"spinal muscular atrophy"[tiab] AND (onasemnogene[tiab] OR nusinersen[tiab] OR risdiplam[tiab] OR "gene therapy"[tiab])Trials: ClinicalTrials.gov studies first submitted in each 12-month window that match the same terms. A query can miss synonyms or catch unrelated papers, so treat counts as approximate.