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 NaliniPublished 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.