Therapeutics in Duchenne muscular dystrophy

Abstract

Summary: Duchenne muscular dystrophy (DMD) is a fatal disorder affecting approximately 1 in 3500 live born males, characterized by progressive muscle weakness. Several different strategies are being investigated in developing a cure for this disorder. Until a cure is found, therapeutic and supportive care is essential in preventing complications and improving the afflicted child's quality of life. Currently, corticosteroids are the only class of drug that has been extensively studied in this condition, with controversy existing over the use of these drugs, especially in light of the multiple side effects that may occur. The use of nutritional supplements has expanded in recent years as researchers improve our abilities to use gene and stem cell therapies, which will hopefully lead to a cure soon. This article discusses the importance of therapeutic interventions in children with DMD, the current debate over the use of corticosteroids to treat this disease, the growing use of natural supplements as a new means of treating these boys and provides an update on the current state of gene and stem cell therapies.

Arquitetura comparativa dos pulmões de camundongos normais e afetados pela Distrofia Muscular de Duchenne

“…Dentre os sinais clínicos observa-se a fraqueza muscular progressiva inicial de cintura pélvica e posteriormente de cintura escapular e com a evolução da doença, observa--se o quadro clínico de insuficiência respiratória e/ou cardíaca, a principal causa "mortis" na DMD (Caromano 1999, Kornegay et al 1999, Strober 2006. Os sinais clínicos respiratórios se exacerbam com o aumento da carga mecânica sustentada a cada movimento respiratório, pela musculatura respiratória já enfraquecida promovendo a redução da complacência pulmonar e da mobilidade da caixa torácica e, por conseguinte hipercapnia, hipoventilação pulmonar, hipoxemia (secundária a hipercapnia) e clearence de secreções (Araujo et al 2004).…”

Duchenne Muscular Dystrophy: Experimental models on Physical Therapy

“…Physical therapy (PT) has been used to reduce muscular, skeletal, cardiac, and vascular abnormalities which develop in association with muscle strength loss (Gaiad et al, 2009). The main objective of such motor therapy is the prevention of muscle contractures and bone deformities (Strober, 2006). However, motor PT approaches have yielded controversial recommendations (Carter et al, 2002) and there is no consensus regarding the type and intensity of physical therapy (Cup et al, 2007).…”

Imagem radiográfica da cavidade torácica de cães Golden Retriever acometidos pela distrofia muscular

“…Como conseqüência, um pulmão fica mais hiperventilado do que o outro. O acúmulo de secreção leva a perda de reflexo do bocejo e tosse, dificultando a higiene brônquica (Strober 2006).…”