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Abstract Details

North African Family With Recessive PYROXD1 Mutation Cause Adult-Onset Limb-Girdle-Type Muscular Dystrophy.
Neuromuscular and Clinical Neurophysiology (EMG)
Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)
016
To describe the first Tunisia/North Africa and the second family in the African continent of adult-onset limb-girdle-type muscular dystrophy caused by biallelic variants in the PYROXD1 gene, which has been recently linked to early-onset congenital myofibrillar myopathy.

45 y/o woman to non-consanguineous parents and without significant past medical history who was referred to neurology clinic for slowly progressing proximal lower extremity weakness. At age 17, she had difficulties in running long distance or participating in gymnastics. Her weakness progressed slowly and at age 35 she experienced trouble going up or down the stairs without holding onto the rails. Her older brother had similar but more severe symptoms, which started at the age of 12. Initially both were diagnosed as limb-girdle muscular dystrophy.

Physical exam showed high arched palate, scoliosis, and joint hypermobility. Limited ankle dorsiflexion to 90 degrees. Normal cranial nerve exam except for palatal weakness and mild neck flexion weakness (5-/5). No ptosis or facial weakness. Mild weakness of proximal upper extremity (deltoid and biceps 5-/5 and triceps 4/5) and normal strength distally. Hip flexion 4/5, knee extension and foot dorsiflexion 5-5/ and normal foot planter flexion. Intact sensation. Reflexes ¼ in the upper and trace in the lower extremities.

Lab showed normal CK (113 IU/ml)) and thyroid function. Unremarkable lumbar spine MRI. 
NCS, needle examination and genetic testing.

Normal NCS. Needle examination showed non-irritable myopathy.

Genetic testing: Clinical exome sequence analysis confirmed pathogenic homozygous c.464 A>G, p.Asn155Ser mutation in PYROXD1 gene.

PYROXD1 gene homozygous mutation is a very rare disease that appears to exhibit a LGMD-like phenotype with childhood or adulthood disease onset.  It has few additional clinical features including scoliosis, high arched palate, nasal speech, and joint hypermobility. Awareness of these clinical features may be helpful to facilitate the diagnosis and lead to the appropriate genetic testing.
Authors/Disclosures
Mohammad I. Alhatou, MD (Alkhor Hospital)
PRESENTER
Dr. Alhatou has nothing to disclose.
Mohamed Ghamoodi Mohamed Ghamoodi has nothing to disclose.
Mohammad I. Alhatou, MD (Alkhor Hospital) Dr. Alhatou has nothing to disclose.