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advancements in prenatal diagnosis and potential fetal therapies for spinal muscular atrophy
Original title: “Advancements in Prenatal Diagnosis and Potential Fetal Therapies for Spinal Muscular Atrophy.”
long term comparative analysis of aav9 mediated gene replacement therapies for spinal muscular atrophy in mice
Original title: “Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice.”
clinical characterization of patients with 5q spinal muscular atrophy types 2 and 3 in brazil a cross sectional observational study
Original title: “Clinical Characterization of Patients With 5q Spinal Muscular Atrophy Types 2 and 3 in Brazil: A Cross-Sectional Observational Study.”
management of spinal muscular atrophy nursing clinical commentary and best practices update
Original title: “Management of spinal muscular atrophy: nursing clinical commentary and best practices update.”
adherence persistence and safety of risdiplam in spinal muscular atrophy a population based cohort study
Original title: “Adherence, Persistence, and Safety of Risdiplam in Spinal Muscular Atrophy: A Population-Based Cohort Study.”
spinal muscular atrophy clinical and genetic aspects and therapeutic alternatives
Original title: “[Spinal muscular atrophy: Clinical and genetic aspects, and therapeutic alternatives].”
feasibility and usefulness of personalised patient reported outcome measures in the therapeutic follow up of adult spinal muscular atrophy patients
Original title: “Feasibility and usefulness of personalised patient-reported outcome measures in the therapeutic follow-up of adult spinal muscular atrophy patients.”
ethics and equity in access to disease modifying therapies and newborn screening for spinal muscular atrophy a scoping review
Original title: “Ethics and equity in access to disease-modifying therapies and newborn screening for spinal muscular atrophy: A scoping review.”
haloperidol induces neuroprotection and enhances neuromuscular function in both murine and human models of spinal muscular atrophy
Original title: “Haloperidol induces neuroprotection and enhances neuromuscular function in both murine and human models of spinal muscular atrophy.”
intersection of spinal muscular atrophy and progressive myoclonic epilepsy syndrome asah1 gene
Original title: “Intersection of Spinal Muscular Atrophy and Progressive Myoclonic Epilepsy Syndrome: ASAH1 Gene.”
Updated dosing offers meaningful clinical benefit
Original title: “FDA approves new high dose Spinraza regimen for SMA”
The FDA rejected the high-dose regimen of Spinraza in September last year due to manufacturing concerns.
Original title: “Biogen Bounces Back With FDA Nod for High-Dose Spinal Muscular Atrophy Drug”
sleep phenotypes and respiratory disturbances in spinal muscular atrophy a systematic review of clinical evidence
Original title: “Sleep phenotypes and respiratory disturbances in spinal muscular atrophy: A systematic review of clinical evidence.”
could an outcome based agreement be operationalized using real world data from the canadian neuromuscular disease registry perspectives from an expert led assessment in spinal muscular atrophy
Original title: “Could an outcome-based agreement be operationalized using real-world data from the Canadian Neuromuscular Disease Registry? Perspectives from an expert-led assessment in spinal muscular atrophy.”
In a letter to European patients, Roche said its decision to end development of “emugrobart” was based on the drug “not consistently” improving muscle growth and motor function in a key study.
Original title: “Roche stops work on experimental SMA drug”
The main beneficiary of Roche’s discontinuation of an investigational spinal muscular atrophy drug is Scholar Rock, which was hobbled by manufacturing concerns at a Novo Nordisk facility last year but is now nearing a potential resolution.
Original title: “Roche Ends Run for Muscular Atrophy Drug, Leaving Door Open for Competitors”
adult sma reach a uk clinical network and real world data collection study for adults living with spinal muscular atrophy
Original title: “Adult SMA REACH: A UK clinical network and real-world data collection study for adults living with spinal muscular atrophy.”
Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies for spinal muscular atrophy, Duchenne muscular dystrophy and Becker muscular dystrophy.
Original title: “MDA 2026: Biogen, Sarepta, Edgewise Address Muscle Disorders”
More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.
Original title: “Biogen builds case for Spinraza successor with fresh data”