Rheumatology
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Better understanding of disease pathophysiology, improved supportive care and availability of disease-specific treatments for some of the mucopolysaccharidosis (MPS) disorders have greatly improved the outlook for patients with MPS disorders. Optimal management of these multisystemic disorders involves a multidisciplinary team and regular, comprehensive follow-up. ⋯ Haematopoietic stem cell transplantation (HSCT) can preserve cognition and prolong survival in very young children with the most severe form of MPS I, and is under investigation for several other MPS disorders. Better tissue matching techniques, improved graft-vs-host prophylaxis and more targeted conditioning regimens have improved morbidity and mortality associated with HSCT.
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To determine whether a high BMI in childhood or early adulthood has a long-term influence on the likelihood of knee pain. ⋯ This study has demonstrated the long-term effects of childhood and early adult obesity and the importance of early intervention programmes to try to reduce weight and maintain weight loss.