The American Journal of Managed Care

Real-World Study Suggests DMD Gene Therapy Is Safe, Effective

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort. Delandistrogene ...
Forbes

The Triumphs And Trials Of Duchenne Gene Therapy

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
New Scientist

First ever inhalable gene therapy for cancer gets fast-tracked by FDA

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...
Good News Network

Successful World First: Baby Treated with Personalized CRISPR Gene Therapy for Rare Disease is Now ‘Thriving’

Dr. Rebecca Ahrens-Nicklas and Dr. Kiran Musunru crafted a bespoke treatment that has successfully corrected the genetic ...
Hosted on MSN

Boy with rare condition amazes doctors after world-first gene therapy

A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy. Oliver Chu has a rare, ...
The New England Journal of Medicine

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency

Migliavacca M, Barzaghi F, Fossati C, et al. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency. Nat Med 2024;30:488-497.
The American Journal of Managed Care

Real-World Data Crucial to Prove the Value of Expensive Cell and Gene Therapies: Fran Gregory, PharmD

As the number of cell and gene therapies expands, it's increasingly important for long-term patient data, explained Fran Gregory, PharmD, MBA, vice president of emerging therapies at Cardinal Health.