Novel gene infusion therapy is a ray of hope for children with DMD
Riley Children’s Health marks a significant milestone in the fight against Duchenne muscular dystrophy (DMD) with the recent administration of gene infusion therapy. Aiming to halt the effects of DMD in pediatric patients, Laurence E. Walsh, MD, a pediatric neurogeneticist and neuromuscular specialist at Riley Children’s, is leading the novel effort.
DMD, a severe genetic muscular disorder primarily affecting boys, leads to muscle degeneration and culminates in paralysis, respiratory complications and premature death. The condition arises from a deficiency of dystrophin, a crucial protein for muscle integrity. While traditional approaches to alleviating DMD involve palliative relief, such as physical therapy and orthopedic interventions, these conventional therapies do not provide a definitive solution. Until recently, with the approval of gene infusion therapy, Riley Children’s has been the first pediatric hospital in the state to administer the newly approved gene infusion therapy, thus offering a new beacon of hope for patients living with the condition.
“Before gene infusion therapy, we’ve never really had a treatment that promises to change the trajectory of the disease to a significant degree,” Dr. Walsh said. “We don’t know the long-term benefits yet, but we know that it has done better than anything else that has come before it.”
Despite the game-changing impacts the new gene infusion therapy provides, the clinical intricacies of DMD still remain profound. In part, the timing of the drug is critical because it is presently only approved to use in children ages 4 to 5. While it’s anticipated that this window will expand if the therapy lives up to its promise, the goal right now is to give kids more time until an even better solution comes to fruition.
“This is a disease I’ve been helping families through for three decades, and it’s pretty neat at this point to have something like this to offer them,” Dr. Walsh said. “It’s incredibly exciting after all this time, to be on the cusp of these gene therapies that I think will make huge differences in children’s lives.”
Looking ahead, the neuromuscular team at Riley Children’s anticipates treating more eligible children with DMD, aiming to preserve their level of function and quality of life overall. So far, Dr. Walsh has administered the drug to two patients at Riley Children’s, and the results look rather promising.
Furthermore, Mara Nitu, MD, chief medical officer at Riley Children’s, also agrees gene infusion therapy is an incredible step toward improving the lives of pediatric patients with DMD, and to be the first hospital in the state to administer the drug is a leading milestone in itself.
“Our mission has always been to provide the best possible care to the patients and families we serve,” Dr. Nitu said. “Being able to offer this therapy underscores our commitment to giving our patients a chance at a brighter future.”
About pediatric neurology
The pediatric neurology program at Riley Children’s is home to Indiana’s first Certified Duchenne Care Center, as recognized by the Parent Project Muscular Dystrophy. In addition, key program highlights include:
- Accredited by the National Association of Epilepsy Centers as a Level 4 Comprehensive Epilepsy Center
- Only facility in Indiana with a Neurofibromatosis Program, Tuberous Sclerosis Program, Neonatal Neurology Program and more
