Projected by some analysts as a future blockbuster, Isembyld is the first “muscle-targeted” treatment for the disease and, in clinical testing, helped improve motor function in people on standard therapies.

Original title: “Scholar Rock SMA drug approved by FDA”

frequency and circumstances of falls events in people living with spinal and bulbar muscular atrophy a cross sectional survey

Original title: “Frequency and Circumstances of Falls Events in People Living With Spinal and Bulbar Muscular Atrophy: A Cross-Sectional Survey.”

integrating lna qpcr and full length smn1 sequencing for precision sma carrier screening large scale validation in 30 849 individuals

Original title: “Integrating LNA-qPCR and full-length SMN1 sequencing for precision SMA carrier screening: large-scale validation in 30,849 individuals.”

spinal muscular atrophy in adult neurology services in india

Original title: “Spinal Muscular Atrophy in Adult Neurology Services in India.”

muscle mri as an imaging biomarker of muscle damage in patients with spinal and bulbar muscular atrophy

Original title: “Muscle MRI as an Imaging Biomarker of Muscle Damage in Patients With Spinal and Bulbar Muscular Atrophy.”

spinal muscular atrophy all babies in england to be screened for rare neuromuscular disease

Original title: “Spinal muscular atrophy: all babies in England to be screened for rare neuromuscular disease.”

The Food and Drug Administration (FDA) is classifying the Spinal Muscular Atrophy newborn screening test system into class II (special controls). The special controls that apply to the device type are identified in this order and will be part of the codified language for classification of the Spinal Muscular Atrophy newborn screening test system. We are taking this action because we have determined that classifying the device into class II will provide a reasonable assurance of safety and effect

Original title: “Medical Devices; Immunology and Microbiology Devices; Classification of the Spinal Muscular Atrophy Newborn Screening Test System”

cost effectiveness of universal newborn screening and disease modifying therapies for spinal muscular atrophy in taiwan

Original title: “Cost-Effectiveness of Universal Newborn Screening and Disease-Modifying Therapies for Spinal Muscular Atrophy in Taiwan.”

After trial flops in spinal muscular atrophy, depression and bipolar disorder—and a costly rare disease drug rejection—Biohaven is undergoing a reset, recasting its former SMA candidate for obesity.

Original title: “Biohaven looks to obesity to bounce back from run of clinical and regulatory failures”

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.”

spinal muscular atrophy clinical and genetic aspects and therapeutic alternatives

Original title: “[Spinal muscular atrophy: Clinical and genetic aspects, and therapeutic alternatives].”

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.”

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.”