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Data from four clinical trials support vamorolone therapy’s safety and efficacy profile in DMD. One study compared 48 weeks of treatment Agamree to prednisone, a corticosteroid, in 121 boys with Duchenne muscular dystrophy.
Duchenne muscular dystrophy is a rare progressive disease that eventually affects all voluntary muscles and involves the heart and breathing muscles in later stages. Life expectancy is estimated to be around 25–26, [18] [59] but this varies. People born with Duchenne muscular dystrophy after 1990 have a median life expectancy of approximately ...
TREAT-NMD (Translational Research in Europe, Assessment and Treatment for NeuroMuscular Disorders) is a global academic network that focuses on advancing research in neuromuscular disorders. [1] It was established in 2007 with its coordination centre at the Newcastle University . [ 2 ]
Beyond academia, Nagaraju is an accomplished entrepreneur, co-founding ReveraGen BioPharma, which develops Vamorolone for inflammatory diseases, including muscular dystrophy, and AGADA BioSciences, which provides preclinical and clinical trial support services for therapeutics targeting rare neuromuscular diseases.
Progressive muscular atrophy (PMA), also called Duchenne–Aran disease and Duchenne–Aran muscular atrophy, is a disorder characterised by the degeneration of lower motor neurons, resulting in generalised, progressive loss of muscle function.
Many affected people will eventually become unable to walk [2] and Duchenne muscular dystrophy in particular is associated with shortened life expectancy. Muscular dystrophy was first described in the 1830s by Charles Bell. [2] The word "dystrophy" comes from the Greek dys, meaning "no, un-" and troph-meaning "nourish". [2]
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