The Circle of Kindness Foundation’s expert council has unanimously approved the initial eight applications to grant children access to the latest medication for treating Duchenne muscular dystrophy. The first injections of the drug are expected to take place in late June – early July.
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is one of four conditions known as dystrophinopathies.
“The Circle of Kindness has provided therapy to more than 400 children with Duchenne muscular dystrophy since 2021. The drugs purchased by the fund, ataluren, eteplirsen, viltolarsen and golodirsen, are suitable for some patients with Duchenne muscular dystrophy and are effective for specific changes in the DMD gene; the drugs must be taken regularly. With the addition of one more drug to the list, the foundation expands the capabilities of Russian doctors to treat Duchenne muscular dystrophy; therapy becomes available to even more children with this disease who are not suitable for other pathogenetic drugs,” the statement says.
Elevidys is a prescription gene therapy used to treat ambulatory children aged 4 through 5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene