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Childhood-Onset Myopathy With Preserved Ambulation Caused by a Recurrent ADSSL1 Missense Variant.

Dipti BaskarKiran PolavarapuVeeramani Preethish-KumarSeena VengalilSaraswati NashiAna TopfAneesha ThomasSai Bhargava SankaDeepak MenonKosha SrivastavaGautham ArunachalBevinahalli N NandeeshHanns LochmüllerAtchayaram Nalini
Published in: Neurology. Genetics (2024)
myopathy with unusual manifestations in this rare disorder. Because the variant c.781G>A (p.Asp261Asn) is the most common mutation among Indian patients similar to other Asian cohorts, this finding could be useful for genetic screening of suspected patients.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • prognostic factors
  • gene expression
  • patient reported outcomes
  • dna methylation
  • pulmonary embolism
  • young adults
  • early onset