Publications by authors named "K M Bushby"
Myostatin is a myokine which acts upon skeletal muscle to inhibit growth and regeneration. Myostatin is endogenously antagonised by follistatin. This study assessed serum myostatin and follistatin concentrations as monitoring or prognostic biomarkers in dysferlinopathy, an autosomal recessively inherited muscular dystrophy.
View Article and Find Full Text PDFWater T2 could predict functional decline in patients with dysferlinopathy.
J Cachexia Sarcopenia Muscle
December 2022
Background: Water T2 (T2 ) mapping is increasingly being used in muscular dystrophies to assess active muscle damage. It has been suggested as a surrogate outcome measure for clinical trials. Here, we investigated the prognostic utility of T2 to identify changes in muscle function over time in limb girdle muscular dystrophies.
View Article and Find Full Text PDFIntroduction/aims: Dysferlinopathy demonstrates heterogeneity in muscle weakness between patients, which can progress at different rates over time. Changing muscle strength due to disease progression or from an investigational product is associated with changing functional ability. The purpose of this study was to compare three methods of strength testing used in the Clinical Outcome Study (COS) for dysferlinopathy to understand which method and which muscle groups were most sensitive to change over time.
View Article and Find Full Text PDFArticle Synopsis
- Corticosteroids like prednisone and deflazacort are effective for improving strength and function in boys with Duchenne muscular dystrophy, but the best dosage and regimen have been unclear.
- This study compared three common corticosteroid regimens (daily prednisone, daily deflazacort, and intermittent prednisone) in 196 boys aged 4 to 7 years over three years, analyzing their efficacy and side effects.
- Results showed that both daily prednisone and daily deflazacort were significantly more effective than intermittent prednisone, with no major differences in effectiveness between the two daily regimens.
Background: Natural history studies in neuromuscular disorders are vital to understand the disease evolution and to find sensitive outcome measures. We performed a longitudinal assessment of quantitative magnetic resonance imaging (MRI) and phosphorus magnetic resonance spectroscopy ( P MRS) outcome measures and evaluated their relationship with function in lower limb skeletal muscle of dysferlinopathy patients.
Methods: Quantitative MRI/ P MRS data were obtained at 3 T in two different sites in 54 patients and 12 controls, at baseline, and three annual follow-up visits.