Respiratory deterioration during growth hormone therapy in a case of congenital nemaline myopathy

Abstract

Congenital nemaline myopathy (CNM) is generally classified as a non-progressive or slowly progressive neuromuscular disease. We describe a boy with CNM and an isolated partial growth hormone (GH) deficiency. From the onset of GH therapy his respiratory capacity deteriorated rapidly. The possible association between this deterioration and GH therapy is discussed.

Muscular dystrophy: Centronucleation may reflect a compensatory activation of defective myonuclei

“…Dystrophic mouse and DMD patient [69,70]; MD patient [21] The sarcomere is abnormally extended Dystrophic chicken [2] Mechanical injury and inflammation of skeletal muscle occur Normal chicken [ 1]; healthy man and normal rat [50]; dystrophic dog [35]; dystrophic mouse [65,66] Growth defect is a common feature of MD Dystrophic mouse [52]; DMD patient [6,15,46,47] Growth hormone treatment may be harmful DMD patient [7]; dwarf patient with nemaline myopathy [34] Striated muscle uninfluenced by bone growth is spared Dystrophic mouse (tongue, ear, nose, esophagus) [22,60,65,66] Reduced load on muscle may be beneficial Dystrophic mouse (immobilization [74], tenotomy [9,32]); dystrophic hamster (suspension [ 16])…”

Muscular Dystrophy: Centronucleation May Reflect a Compensatory Activation of Defective Myonuclei

“…(1) A number of myotubes or immature myofibers are unable to clear possible hurdles to maturation with resultant growth defects becoming evident during the first 2 weeks of life [58]. (2) Most myofibers which have survived the initial period do not grow but those that do demonstrate a varied growth capacity and consequent- [69,70]; MD patient [21] The sarcomere is abnormally extended Dystrophic chicken [2] Mechanical injury and inflammation of skeletal muscle occur Normal chicken [1]; healthy man and normal rat [50]; dystrophic dog [35]; dystrophic mouse [65,66] Growth defect is a common feature of MD Dystrophic mouse [52]; DMD patient [6,15,46,47] Growth hormone treatment may be harmful DMD patient [7]; dwarf patient with nemaline myopathy [34] Striated muscle uninfluenced by bone growth is spared Dystrophic mouse (tongue, ear, nose, esophagus) [22,60,65,66] Reduced load on muscle may be beneficial Dystrophic mouse (immobilization [74], tenotomy [9,32]); dystrophic hamster (suspension [16])…”