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Phenotype profile

spinal muscular atrophy

SaudiVarKB evidence summary derived from retained literature mentions.

38Phenotype mentions
38Publications
14Associated gene records
8Associated variant records

Associated gene records

Co-mentioned in the same publications
GeneIdentifier / contextArticlesMentions
SMN1HGNC:111171111
SMN2HGNC:1111866
NAIPHGNC:763444
CFTRHGNC:188422
IGHMBP2HGNC:554222
PAHHGNC:858211
PTENHGNC:958811
NEBHGNC:772011
PLS3HGNC:909111
PRMT1HGNC:518711
EXOSC9HGNC:913711
EXOSC3HGNC:1794411
EXOSC8HGNC:1703511
RBM7HGNC:990411

Associated variant records

Co-mentioned in the same publications
VariantIdentifier / contextArticlesMentions
c.48_55dupGGATTCCGc.48_55dupGGATTCCG11
c.662C>Tc.662C>T11
p.Pro221Leup.Pro221Leu11
rs143838139rs14383813911
rs121909192rs12190919211
c.41T>Cc.41T>C11
p.Leu14Prop.Leu14Pro11
c.481C>Tc.481C>T11

Associated population records

Co-mentioned in the same publications
PopulationIdentifier / contextArticlesMentions
Saudi Arabia33
Saudi Arabia22
Population record11
Saudi ArabiaCohort 11011
Population recordCohort 2611
Saudi ArabiaCohort 4611
Saudi ArabiaCohort 1,50211
Saudi ArabiaCohort 1411
Population recordCohort 3411
Saudi ArabiaCohort 1611
Saudi ArabiaCohort 2511
Saudi ArabiaCohort 011
Population recordCohort 12611
Saudi ArabiaCohort 5211
Saudi Arabia · Eastern Province11
Saudi Arabia · RiyadhCohort 8411
Saudi ArabiaCohort 9811
Saudi Arabia · RiyadhCohort 12111

Supporting publications

38 records
  1. 2026Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.Nature medicine1 mentions
  2. 2025Onasemnogene Abeparvovec is Safe in Hemolytic Disease of the Newborn: A Case Report.Neuropediatrics1 mentions
  3. 2025Anaesthetic management of spinal muscular atrophy in a patient with pneumothorax: a case report.JPMA. The Journal of the Pakistan Medical Association1 mentions
  4. 2025Unlocking the potential: advancements and applications of gene therapy in severe disorders.Annals of medicine1 mentions
  5. 2025Risdiplam in Presymptomatic Spinal Muscular Atrophy.The New England journal of medicine1 mentions
  6. 2025Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives.Current genomics1 mentions
  7. 2025Real-world evidence of Nusinersen treatment for patients with spinal muscular atrophy in the Kingdom of Saudi Arabia: Initial insights from the Saudi national spinal muscular atrophy program.Saudi medical journal1 mentions
  8. 2024Life-Saving Treatments for Spinal Muscular Atrophy: Global Access and Availability.Neurology. Clinical practice1 mentions
  9. 2024A Case of a Newborn With Nemaline Myopathy From Al-Qunfudhah City, Saudi Arabia.Cureus1 mentions
  10. 2024Unveiling the genetic tapestry: Rare disease genomics of spinal muscular atrophy and phenylketonuria proteins.International journal of biological macromolecules1 mentions
  11. 2024Spinal muscular atrophy genetic epidemiology and the case for premarital genomic screening in Arab populations.Communications medicine1 mentions
  12. 2023Identifying Clinical and Genetic Characteristics of Spinal Muscular Atrophy Patients and Families in Saudi Arabia.Cureus1 mentions
  13. 2023Studying carrier frequency of spinal muscular atrophy in the State of Qatar and comparison to other ethnic groups: Pilot study.Molecular genetics & genomic medicine1 mentions
  14. 2023Nucleolar reorganization after cellular stress is orchestrated by SMN shuttling between nuclear compartments.Nature communications1 mentions
  15. 2022Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.Molecular neurobiology1 mentions
  16. 2022Spinal muscular atrophy carrier frequency in Saudi Arabia.Molecular genetics & genomic medicine1 mentions
  17. 2022Medical treatment of patients with hypertrophic cardiomyopathy: An overview of current and emerging therapy.Archives of cardiovascular diseases1 mentions
  18. 2021Spinal Muscular Atrophy with Respiratory Distress<br /> Type 1: A Novel Variant of IGHMBP2 Gene.Journal of the College of Physicians and Surgeons--Pakistan : JCPSP1 mentions
  19. 2020Spinal Muscular Atrophy and Communicating Hydrocephalus: A Novel or a Well-Established Rare Association?Cureus1 mentions
  20. 2019Evaluation of the role of an antioxidant gene in NSC-34 motor neuron-like cells as a model of a motor neuron disease.Folia morphologica1 mentions
  21. 2018Electrochemical immunosensors for the detection of survival motor neuron (SMN) protein using different carbon nanomaterials-modified electrodes.Biosensors & bioelectronics1 mentions
  22. 2018Variants in EXOSC9 Disrupt the RNA Exosome and Result in Cerebellar Atrophy with Spinal Motor Neuronopathy.American journal of human genetics1 mentions
  23. 2018Carbon nanofiber-based multiplexed immunosensor for the detection of survival motor neuron 1, cystic fibrosis transmembrane conductance regulator and Duchenne Muscular Dystrophy proteins.Biosensors & bioelectronics1 mentions
  24. 2017Spinal Muscular Atrophy and Ependymoma.Saudi journal of medicine & medical sciences1 mentions
  25. 2015PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.Molecular therapy : the journal of the American Society of Gene Therapy1 mentions
  26. 2013Type III spinal muscular atrophy mimicking muscular dystrophies.Pediatric neurology1 mentions
  27. 2013A homozygous double mutation in SMN1: a complicated genetic diagnosis of SMA.Molecular genetics & genomic medicine1 mentions
  28. 2010Infantile spinal muscular atrophy with respiratory distress type 1: a case report.Journal of child neurology1 mentions
  29. 2010Permissibility of prenatal diagnosis and abortion for fetuses with severe genetic disorder: type 1 spinal muscular atrophy.Annals of Saudi medicine1 mentions
  30. 2005Spinal muscular atrophy carrier screening by multiplex polymerase chain reaction using dried blood spot on filter paper.Annals of human genetics1 mentions
  31. 2003Molecular analysis of the spinal muscular atrophy and neuronal apoptosis inhibitory protein genes in Saudi patients with spinal muscular atrophy.Saudi medical journal1 mentions
  32. 2001Gene deletion patterns in spinal muscular atrophy patients with different clinical phenotypes.Journal of biomedical science1 mentions
  33. 1999Application of DNA-based tests for diagnosis of spinal muscular atrophy in Saudi Arabia.Eastern Mediterranean health journal = La revue de sante de la Mediterranee orientale = al-Majallah al-sihhiyah li-sharq al-mutawassit1 mentions
  34. 1998Molecular analysis of the SMN and NAIP genes in Saudi spinal muscular atrophy patients.Journal of the neurological sciences1 mentions
  35. 1996Childhood neuromuscular disorders: a decade's experience in Saudi Arabia.Annals of tropical paediatrics1 mentions
  36. 1993A community survey of neurological disorders in Saudi Arabia: the Thugbah study.Neuroepidemiology1 mentions
  37. 1992Werdnig Hoffman's disease (spinal muscular atrophy type I): A clinical study of 25 Saudi nationals in Al-Khobar.Annals of Saudi medicine1 mentions
  38. 1991Genetically determined neurodegenerative disorders: experiences in Saudi Arabia.Annals of tropical paediatrics1 mentions