Kazuki Sawamoto, Yasuyuki Suzuki, William G Mackenzie, Mary C Theroux, Christian Pizarro, Hiromasa Yabe, Kenji E Orii, Robert W Mason, Tadao Orii, Shunji Tomatsu
INTRODUCTION: Morquio A syndrome is characterized by a unique skeletal dysplasia, leading to short neck and trunk, pectus carinatum, laxity of joints, kyphoscoliosis, and tracheal obstruction. Cervical spinal cord compression/inability, a restrictive and obstructive airway, and/or bone deformity and imbalance of growth, are life-threatening to Morquio A patients, leading to a high morbidity and mortality. It is critical to review the current therapeutic approaches with respect to their efficacy and limitations...
2016: Expert Opinion on Orphan Drugs