PPMD ha creado un video para que los niños expliquen Duchenne y varias presentaciones sobre cuidados para familias de habla hispana.

PPMD ha creado un video para que los niños expliquen Duchenne y varias presentaciones sobre cuidados para familias de habla hispana.

PPMD ha creado un video para que los niños expliquen Duchenne y varias presentaciones sobre cuidados para familias de habla hispana.

PPMD ha creado un video para que los niños expliquen Duchenne y varias presentaciones sobre cuidados para familias de habla hispana.

On April 20, 2021, PPMD’s Pat Furlong & Eric Camino were joined by Drs. Nagaraju and Shieh for an interactive panel discussion on the lessons learned in Gene Therapy, current challenges for our community, and what the future holds for these promising treatments.

In March 2022, Dr. Francessco Muntoni, MD, Great Ormond Street Institute of Child Health in London and Dr. Carsten Bonnemann, MD, National Institute of Health, brought together four companies with ongoing microdystrophin gene therapy programs to discuss recent Serious Adverse Events (SAEs). The objective of the working group is to share data and better understand the cause of these SAEs and how we may be able to treat those patients with genetic variants that may be at higher risk for an SAE.

This February 2021 PGTME webinar provides an overview of neutralizing antibodies (the antibodies created when the immune system has seen this virus and developed resistance) and the challenges they create in the delivery of gene therapy.

Barry Byrne, MD, PhD joins PPMD's Pat Furlong for an Innovation Conversation on antibodies in gene therapy on December 21, 2023.

An additional challenge with micro-dystrophin gene replacement is an immune response to the newly produced micro-dystrophin protein.