Sarepta's recent announcement of the U.S. Food and Drug Administration's approval for ELEVIDYS to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a significant advancement in Duchenne muscular dystrophy (DMD) treatment. DMD, a severe form of inherited muscular dystrophies, results in muscle degeneration and weakness due to mutations in the dystrophin gene.
Viral-mediated gene therapy, like ELEVIDYS, offers hope to DMD patients. Sarepta's leadership in DMD treatment underscores the importance of this milestone for the Duchenne community and the field of gene therapy.
Doug Ingram, president and CEO of Sarepta, rightly emphasized the impact of this expansion, calling it a win for science and a promising moment for gene therapy. This development not only highlights progress in DMD treatment but also signifies a step forward in the application of gene therapy to address genetic disorders.
This expansion of the ELEVIDYS label marks a positive shift in the landscape of DMD treatment, offering new possibilities for patients and families affected by this debilitating condition.
"Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science," said Doug Ingram, president and chief executive officer, Sarepta.
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