DMD – THERAPY

Symptomatic therapy of Duchenne Muscular Dystrophy (DMD)

DMD certainly has a remarkable position amongst all hereditary muscle disorders. Noteworthy is its relatively high incidence as a " rare disease " , its X-chromosomal mode of inheritance, the high frequency of new mutations , as well as its relatively rapid and fatal course. For a century the disorder was considered to be untreatable. Only 40 years ago the patients' " natural " age at death was...

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Prosthodontics, Scranton, Pennsylvania. ter of routine. However, in anterior implant -supported restorations, the successful achievement of the aesthetic goal can often be elusive. The task is made especially arduous when the potential implant recipient site lies within the lip perimeter and is compromised by significant hard and soft tissue defects and/or inharmonious occlusal combinations. Un...

T.I.1 Potential for gene therapy in DMD

(population 5.5 million). We conducted a screening program in the largest neuromuscular clinic, and respiratory center in Denmark, as we hypothesised that the condition may be overlooked. The inclusion criteria were age (over 15 years), unexplained hyper-CK-aemia and myopathy, unclassified Limb-girdle muscular dystrophy (LGMD) and unexplained, restrictive respiratory insufficiency. We went thro...

Identification of serum protein biomarkers for utrophin based DMD therapy

Despite promising therapeutic avenues, there is currently no effective treatment for Duchenne muscular dystrophy (DMD), a lethal monogenic disorder caused by the loss of the large cytoskeletal protein, dystrophin. A highly promising approach to therapy, applicable to all DMD patients irrespective to their genetic defect, is to modulate utrophin, a functional paralogue of dystrophin, able to com...

DMD # 10249 1 Enzyme