Lurie Kids’s Hospital administers first gene remedy for Duchenne muscular dystrophy in Illinois

On March 27, 2024, Ann & Robert H. Lurie Kids’s Hospital of Chicago handled its first affected person with ELEVIDYS (delandistrogene moxeparvovec-rokl), the primary gene remedy for Duchenne muscular dystrophy – a uncommon, genetic illness characterised by progressive muscle injury and weak point. Lurie Kids’s is the primary in Illinois to manage this remedy after ELEVIDYS obtained U.S. Meals and Drug Administration (FDA) approval in June 2023.

Developed by Sarepta Therapeutics, ELEVIDYS is authorized for the remedy of Duchenne muscular dystrophy (DMD) in ambulatory sufferers aged 4 via 5 years with a confirmed mutation within the DMD gene.

Our crew at Lurie Kids’s has had encouraging expertise with this gene remedy for Duchenne via our energetic participation in scientific trials. Over the previous two years, we’ve got handled three boys with DMD with ELEVIDYS as half of a bigger scientific trial, and it is gratifying to see that their muscle energy and performance stabilized. With out gene remedy, we might anticipate to see ongoing deterioration in muscle perform in these boys. This remedy just isn’t a remedy, and sadly it can’t reverse earlier muscle injury, however we anticipate that we are able to decelerate the illness sufficient for science to step in and provide new therapies. That is the start of a really thrilling journey.”

Nancy Kuntz, MD, Director of Muscular Dystrophy Affiliation Care Heart at Lurie Kids’s and Professor of Pediatrics and Neurology at Northwestern College Feinberg Faculty of Drugs

Duchenne happens in roughly one in each 3,500-5,000 new child males worldwide. It’s attributable to mutations within the dystrophin gene that result in an absence of dystrophin protein, which acts as a shock absorber when muscle tissue transfer. The primary delicate indicators of DMD could seem throughout infancy. Muscle weak point turns into more and more noticeable between the ages of three and 5 years with the prognosis being sometimes made round these ages. Most kids dwelling with Duchenne use a wheelchair by age 13 years. The main causes of loss of life in people with Duchenne are respiratory or cardiac failure, which generally happens when sufferers are of their mid-20s/30s.

Mason Flessner, now an lively 6-year-old, was one of many scientific trial members at Lurie Kids’s who obtained ELEVIDYS about eight months in the past. He now is ready to run sooner, climb stairs extra simply and even soar – one thing he could not do beforehand. His little brother, 3-year-old Dawson, who additionally has Duchenne, is ready till he’s sufficiently old to qualify for gene remedy.

“ELEVIDYS has been life-changing for Mason, and it has given us hope and optimism that Duchenne is not a deadly prognosis,” stated Dan Flessner, Mason’s father. “Due to analysis, gene remedy now provides us a pathway to a remedy. With a lot progress already, it is not a pipedream anymore.”

ELEVIDYS is run as a one-time intravenous infusion. The gene remedy addresses the foundation genetic reason behind Duchenne by delivering a gene that codes for a shortened type of dystrophin to muscle cells, referred to as ELEVIDYS-dystrophin. As a result of dystrophin gene is the biggest recognized human gene, scientists engineered a shortened model of the gene that would match inside present gene remedy supply applied sciences and nonetheless retain key practical data. The remedy’s accelerated approval relies on a rise in ELEVIDYS-dystrophin protein expression in skeletal muscle cells.

“Throughout the nation, for the reason that FDA approval, ELEVIDYS has solely been administered just a few occasions and we’re very excited to be the primary website in Illinois to manage it,” stated Abigail Schwaede, MD, one of many neuromuscular physicians on Mason’s care crew at Lurie Kids’s and Assistant Professor of Pediatrics at Northwestern College Feinberg Faculty of Drugs. “This remedy has huge potential to enhance the standard of life and long-term outcomes for boys with Duchenne muscular dystrophy.”