Publications by authors named "L-J Mah"
Article Synopsis
- Vamorolone, a glucocorticoid receptor agonist, was tested to assess its effectiveness and safety over 48 weeks compared to prednisone in children with Duchenne muscular dystrophy (DMD).
- A double-blind clinical trial involved 121 participants aged 4 to under 7 years, receiving varying doses of vamorolone and prednisone, with improvements monitored in motor skills and growth.
- Results indicated that vamorolone (6 mg/kg/day) maintained motor skill improvements over 48 weeks, with significant growth benefits seen after participants switched from prednisone to vamorolone.
Article Synopsis
- 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.
