Publications by authors named "P-M Mah"
Background: A growing number of patients with tetralogy of Fallot develop left ventricular systolic dysfunction and heart failure, in addition to right ventricular dysfunction. Although cardiac resynchronization therapy (CRT) is an established treatment option, the effect of CRT in this population is still not well defined. This study aimed to investigate the early and late efficacy, survival, and safety of CRT in patients with tetralogy of Fallot.
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- The study evaluated vamorolone, a novel anti-inflammatory drug, in 48 boys aged 4-7 with Duchenne muscular dystrophy (DMD) to determine the optimal dosage and effectiveness.
- Conducting a 24-week trial with varying doses (0.25, 0.75, 2.0, and 6.0 mg/kg/d), researchers found that the 2.0 mg/kg/d dose significantly improved muscle function without the common side effects associated with glucocorticoids.
- Results indicated that vamorolone was safe and well-tolerated, showing potential benefits in bone health and lower risk of adrenal suppression and insulin resistance compared to traditional glucocorticoid treatments.
Objectives: To investigate motor function associations with age, sex, and repeats among participants with early-onset facioscapulohumeral muscular dystrophy (FSHD) type 1 as defined by weakness onset before 10 years of age.
Methods: We collected standardized motor assessments, including manual muscle testing (MMT), quantitative muscle testing, functional motor evaluations, and clinical severity scores (CSSs), at 12 Cooperative International Neuromuscular Research Group centers. To measure associations, we used linear regression models adjusted for sex, evaluation age, age at onset of weakness, and repeats.