Adrenal suppression from vamorolone and prednisone in Duchenne muscular dystrophy: results from the phase 2b clinical trial

A Ahmet, R Tobin, UJ Dang, R Rooman… - The Journal of …, 2024 - academic.oup.com
Context Vamorolone, a novel “dissociative” steroid, demonstrated similar efficacy in muscle
function relative to prednisone 0.75 mg/kg/day but improved linear growth and bone …

[HTML][HTML] Xp21 contiguous gene deletion syndrome presenting as Duchenne muscular dystrophy and glycerol kinase deficiency associated with intellectual disability …

A Pizza, E Picillo, ME Onore, M Scutifero… - Acta …, 2023 - ncbi.nlm.nih.gov
The contiguous gene deletion syndromes (CGDS) are rare genomic disorders resulting from
the deletion of large segments of DNA, manifested as the concurrence of apparently …

Glycerol Kinase Deficiency

S Mettananda, V Arunath - Genetic Syndromes: A Comprehensive …, 2023 - Springer
Springer MRW: [AU:, IDX:] Page 1 Glycerol Kinase Deficiency Sachith Mettananda and
Visvalingam Arunath Contents Other Names .............................................................................. 1 …