Research highlights, computational discoveries, and platform updates. Each post documents a specific finding with full methodology and source citations. Comment on findings and join the discussion.
The U.S. Food and Drug Administration (FDA) is expected to decide in the coming months whether or not to approve apitegromab, an experimental muscle-strengthening agent designed…
What was reported Gerstner, Wittig, Menedo, Ruwald, Sumner, Mentis, Pellizzoni and colleagues (Leipzig, Columbia, Johns Hopkins, Ulm, DZNE) reported at the **5th International…
What was reported Vrettou, Müller, Zetzsche and Wirth (University of Cologne) presented multi-organ proteomics in the Taiwanese SMA mouse model + ASO-treated controls at the **5th…
What was reported Donadio, Mickle, Ostrowski, Mentis, Friedlander, Capogrosso and colleagues (Pittsburgh + Carnegie Mellon + Columbia) reported at the **5th International…
What was reported Coppejans, Vanhauwaert and colleagues (argenx, Edinburgh, Harvard) presented preclinical data at the 5th International Scientific Congress on SMA (Budapest 2026)…
What was reported At the 5th International Scientific Congress on SMA (Budapest, 11–14 March 2026), Crawford, Sansone, Hagenacker and colleagues from Biogen presented Phase 1…
Cross-database analysis of FDA-approved drugs against the HDAC6 mechanism validated by Osseni et al. (Brain 2026) for SMA identifies Givinostat (Duvyzat, Italfarmaco) as the…
FeaturedScholar Rock announced 8 May 2026 that the U.S. Food and Drug Administration has accepted its Biologics License Application (BLA) for apitegromab. The Prescription Drug User Fee…
Kannan et al. (Brain Communications, 2026) demonstrate that pharmacological inhibition of c-Jun NH2-terminal kinase (JNK) prevents motor neuron degeneration in SMA models —…
Menduti et al. (Experimental and Molecular Medicine, 2026) report that haloperidol — a D2 dopamine receptor antagonist FDA-approved since 1967 for psychosis — induces…
On 23 April 2026 the European Medicines Agency Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Itvisma , a Novartis intrathecal AAV9-delivered…
Osseni et al. (Brain, 2026) report that combining SMN2 splice modifiers (such as antisense oligonucleotides inducing exon 7 inclusion) with systemic HDAC6 inhibition strongly…
Children with spinal muscular atrophy (SMA) who have two or fewer copies of the SMN2 gene — a feature typically associated with more severe disease — are more likely to start…
A panel of experts has developed the first U.K. national consensus recommendations for preventing and managing hip problems in children with spinal muscular atrophy (SMA), aiming…
Headline AlphaFold-3 batch 04.05 returned high-confidence iPTM scores (0.80-0.83) for four key protein-pair interactions previously flagged as structurally uncertain. The…
FeaturedA new high-dose regimen of Spinraza (nusinersen) was recently approved in the U.S. as a treatment for spinal muscular atrophy (SMA), with clinical trial data showing a safety…
TL;DR AlphaFold-3 batch 20260429_02804 (33 protein-protein interface jobs, 5 model seeds each) ingested into the platform. First structural validation of the LIMK2×CFL2 disease…
FeaturedSpinal muscular atrophy (SMA) is more common in wealthier countries across the globe, likely reflecting increased access to diagnostic tools, newborn screening, and modern…
A European Medicines Agency (EMA) panel recommended that the one-time gene therapy Itvisma (onasemnogene abeparvovec-brve) be approved for patients with spinal muscular atrophy…
3-LLM consensus validator now PRODUCTION (v2) The Spark Chai-1 saturator surfaces protein-ligand pair predictions continuously, and a Modal serverless A100 backend re-runs each…
FeaturedThree-backend research compute fleet now live The SMA research platform now operates three independent GPU compute backends in parallel, each running pharma-relevant workloads…
FeaturedNewborn screening (NBS) for spinal muscular atrophy (SMA) is widely supported among families whose babies were screened, SMA families and adults living with the disease,…
Targeting a stress-activated signaling pathway may offer a new way to treat spinal muscular atrophy (SMA), either on its own or in combination with existing therapies. Researchers…
The antipsychotic medication haloperidol may help treat spinal muscular atrophy (SMA) by increasing SMN protein levels and enhancing protective effects in nerve cells, a study…
A review study found that the neuromuscular junction (NMJ), the site where nerves connect to the muscles they control, fails to mature normally soon after birth in people with…
Treatment with Evrysdi (risdiplam) improved motor function and quality of life in children and adults with spinal muscular atrophy (SMA), a real-world study in Hong Kong shows.…
Far-field potentials (FFPs), electrical signals recorded with surface electrodes after nerve stimulation, may help track disease progression in people with spinal muscular atrophy…
A home-based respiratory physiotherapy program focused on breathing exercises and airway clearance techniques can significantly improve quality of life for young people with…
Surgery to implant magnetically controlled growing rods (MCGRs) is a safe and effective option for children with spinal muscular atrophy (SMA) type 1 and scoliosis, according to a…
Whole-body quantitative MRI, or qMRI, can track long-term subtle muscle changes throughout the body among adults with spinal muscular atrophy (SMA), despite treatment and clinical…
Sleep issues, common in people with spinal muscular atrophy (SMA), may be driven not only by nighttime breathing problems linked to weak respiratory muscles but also by changes in…
The news The FDA has approved a higher-dose nusinersen (Spinraza) regimen for SMA, shortening the time-to-maintenance-dose and potentially increasing CSF exposure in…
FeaturedThe finding Polish cohort (n = 38, 1-year follow-up, BMC Pediatrics ): higher BMI trended toward better gross motor scores in SMA children; fine motor showed inverse pattern in…
The finding Systematic review of 20 studies (SMA News Today 2026-04-10, Sleep Medicine ) shows SMA sleep disturbances are driven by a dual mechanism : respiratory muscle weakness…
The finding German cohort of Spinraza-treated adults (n = 9, age 31-66, median 4.5-year follow-up) shows quantitative whole-body MRI detects **muscular fat fraction (mFF) changes…
FeaturedThe finding A German cohort (n = 30+ SMA infants treated with nusinersen or onasemnogene abeparvovec) shows that swallowing development remains delayed compared with healthy…
FeaturedThe thesis Fasudil alone is a single-mechanism cytoskeletal rescue. The 2026 SMA Congress (Budapest) and the Cure SMA 2025 meeting together handed the field two orthogonal axes…
FeaturedThe finding Gerstner et al. (Leipzig, Brain 2025, PMID 40585211 ) and Cottam et al. (Brain Pathology 2025, 35(6):e70025) converge on one uncomfo...
The headline Scholar Rock's apitegromab (SRK-015, selective latent myostatin inhibitor) hit its Phase 3 primary endpoint in the SAPPHIRE trial for nonambulatory Type II/III SMA…
FeaturedThe headline At the 2026 SMA Congress (Budapest, O38), Thomas Crawford (Johns Hopkins) presented Phase 1 interim data and the Phase 3 design for salanersen (BIIB115) — Biogen's…
FeaturedThe new orthogonal axis At the 2026 SMA Congress (Budapest), Ilaria Bruno (Viero lab, CNR Trento, O24) presented *"Rescuing translation defects as a new SMN-independent…
FeaturedThe opportunity Session 3 at the 2026 SMA Congress (Budapest) put metabolism and redox back on the map. Vrettou et al. (Cologne, O16, bioRxiv 2026-01-09) show SMN deficiency…
FeaturedWhat the 2026 SMA Congress told us The 5th International Scientific Congress on SMA (Budapest, 11–14 March 2026) reframed the agrin-LRP4-MuSK-DOK7-RAPSN NMJ axis as the #1 unmet…
FeaturedTL;DR 50 completed MD trajectories (47.8 GB total) had been sitting on the cluster without analysis. We ran MDAnalysis -based backbone/ligand/contact/pocket-retention analysis on…
Paradigm correction (the second one in a week) After three new DiffDock batches and an orphan-trajectory analysis, the 4-AP story is clearer — and smaller — than either of our…
FeaturedTL;DR We generated 52 AAV9 VP1 capsid variants with redesigned receptor-binding loops VR-V (490-506) and VR-VIII (581-597) using RFdiffusion (backbone) and ProteinMPNN (sequence).…
FeaturedSummary (published as negative result with same rigor as positive ones) We systematically modified Fasudil (approved ROCK inhibitor; DILI 0.28, BBB 0.93) across 9…
Paradigm correction Initial (wrong) framing : "Does 4-AP modulate SMN2 splicing or replace nusinersen?" Answer (after MD evidence + Simon input) : NO. 4-AP does not bind SMN2…
TL;DR Cas-OFFinder off-target analysis on 6 candidate SMN2 base-editing gRNAs (hg38, up to 4 mismatches) returned 2,097 total hits . Three key findings: One guide must be…
FeaturedTL;DR Seven additional molecules from the PocketXMol DFG-out Type II library passed our LIMK2 selectivity filter overnight (2026-04-09 → 04-10). Combined with the previous…
Featured