Publications by authors named "Migvis Monduy"
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
- Long-term use of corticosteroidal anti-inflammatory drugs can negatively impact patient quality of life, highlighting the need for safer alternatives.
- The study tested vamorolone, a new type of dissociative steroid, for its effectiveness and safety in boys aged 4 to under 7 with Duchenne muscular dystrophy (DMD) over a 24-week period.
- Results showed that vamorolone (6 mg/kg) significantly improved motor function compared to placebo, while the safety profile was potentially better than traditional corticosteroids.
Article Synopsis
- The study investigated the effectiveness of fosmetpantotenate in improving symptoms and stabilizing disease progression for patients with Pantothenate kinase-associated neurodegeneration (PKAN), a condition with no approved treatments.
- Conducted over 24 weeks, the randomized, double-blind, placebo-controlled trial included 84 patients aged 6 to 65 with specific genetic mutations, comparing the effects of a 300 mg oral dose of fosmetpantotenate against a placebo.
- Results showed that fosmetpantotenate was safe; however, it did not demonstrate a significant improvement in patient function as measured by the PKAN-Activities of Daily Living scale.
