N. Jeyaganeshan¹, Nirubaa Umasankar², Rojitha Sivasupiramaniam¹
1- Teaching Hospital Jaffna, Sri Lanka
2- Faculty of Medicine, University of Jaffna, Sri Lanka
Abstract Body
Introduction: Emery-Dreifuss muscular dystrophy (EDMD) is a rare genetic disorder characterized by slowly progressive muscle weakness, joint contractures, and cardiac dysfunction. Within this spectrum, autosomal dominant EDMD Type 2, caused by mutations in the LMNA gene (encoding lamin A/C). Symptoms normally appear in the second decade of life or later.
Case presentation: A 2-year-old boy , the first-born child of a non-consanguineous couple presented with the features of isolated gross motor delay . The examination of lower limbs revealed pseudohypertrophy of the calves and positive Gowers sign, along with generalized hypotonia and reduced reflexes. There were no joint contractures. The abdominal examination did not reveal hepatomegaly or splenomegaly . Cardiovascular system was clinically normal. Investigations revealed persistently high creatine phosphokinase of 500-800 U/L. Venous blood gas and liver function tests, including transaminases, alkaline phosphatase, gamma-glutamyl transferase were normal. Abdominal ultrasonography , electrocardiography (ECG) and initial echocardiography were normal. A technically limited electromyogram (due to poor cooperation) does not showed any evidences of myopathy or for an anterior horn cell disease. The whole exome sequencing identified a pathogenic heterozygous variant in the LMNA gene (NM¬¬_170707 .4): c.745C>T, (p. Arg249Trp) confirming autosomal dominant EDMD type 2. The child is currently managing with regular cardiac monitoring (ECG, echocardiography) and limb physiotherapy .
Conclusions: We report an extremely rare childhood presentation of Emery-Dreifuss muscular dystrophy type 2. This case report highlights the importance of evaluating peripheral hypotonia and the usefulness of whole exome sequencing in establishing an accurate diagnosis when relatively uncommon diseases are suspected.