The drug has been recommended for use on the NHS in England, Wales and Northern Ireland to treat the rare muscle-wasting disorder in those aged four years and older, with approximately 1,700 patients expected to benefit from the endorsement in England alone.
Estimated to affect one in every 3,500 male births globally, DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles. Major milestones of the disease are the loss of ambulation and self-feeding, the start of assisted ventilation, and the development of cardiomyopathy…
