In the treatment of Duchenne
muscular dystrophy (DMD)

Illuminate a potential
path forward

Pfizer’s investigational gene therapy is being evaluated in clinical trials for the treatment of DMD and is not FDA approved.^1-4

Gene therapy could provide
a new direction^1,5

Gene therapy is a type of genetic medicine that introduces a functional gene into cells, providing them with the ability to produce a missing or nonfunctional protein.⁶

Varied disease progression
among patients with DMD

Over time, the way symptoms manifest and impact the body can vary for each patient.^7,8

Explore the signs

Tools for assessing
motor function

There are several methods for evaluating motor function in ambulatory boys with DMD.

Learn about measures of disease

Coordinated care for individualized needs

A multidisciplinary care approach continues to be crucial in addressing patient clinical needs.⁹

Find out more

Ongoing DMD research

Several different approaches to genetic medicine are currently being researched in DMD.^1,2

Review the research

Gene therapy’s potential

The goal of gene therapy for DMD is to slow or stabilize muscle degeneration, but there are considerations.^1,5

Discover the potential

Gene therapy is an evolving science

Research is ongoing to understand Pfizer's investigational gene therapy for DMD, including safety considerations, immune response, efficacy, durability, and post-treatment considerations.^1-3,10,11

If Pfizer's gene therapy for DMD is FDA approved, patients will be monitored in the short and long term following treatment.

Explore considerations

Download an English or Spanish language resource regarding DMD
and the potential of gene therapy below

Thumbnail of downloadable English resource to learn more about DMD and potential of gene therapy

Download English PDF

Thumbnail of downloadable Spanish resource to learn more about DMD and potential of gene therapy

Descárguese el PDF en español

Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7(1):13.
Fortunato F, Rossi R, Falzarano MS, Ferlini A. Innovative therapeutic approaches for Duchenne muscular dystrophy. J Clin Med. 2021;10(4):820.
A study to evaluate the safety and tolerability of PF-06939926 gene therapy in Duchenne muscular dystrophy. Published January 10, 2022. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/NCT03362502?term=NCT03362502
Gene and Cell Therapy FAQs. American Society of Gene & Cell Therapy. Accessed November 11, 2022. https://asgct.org/education/more-resources/gene-and-cell-therapy-faqs
Kamdar F, Garry DJ. Dystrophin-deficient cardiomyopathy. J Am Coll Cardiol. 2016;67(21):2533-2546.
Gene Therapy Basics. American Society of Gene & Cell Therapy. Accessed December 7, 2023. https://patienteducation.asgct.org/gene-therapy-101/gene-therapy-basics
Muntoni F, Domingos J, Manzur AY, et al. Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy. PLoS One. 2019;14(9):1-21.
Muntoni F, Guglieri M, Mah JK, et al. Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial. PLoS One. 2022;17(8):1-15.
Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267.
A gene transfer therapy study to evaluate the safety and efficacy of SRP-9001 in participants with Duchenne muscular dystrophy (DMD) (EMBARK). Published December 16, 2021. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/NCT05096221
Microdystrophin gene transfer study in adolescents and children with DMD (IGNITE DMD). Updated August 24, 2021. Accessed November 11, 2022. https://clinicaltrials.gov/ct2/show/NCT03368742?term=NCT03368742

In the treatment of Duchenne muscular dystrophy (DMD)

Illuminate a potential path forward

Gene therapy’s potential

Gene therapy is an evolving science

References

In the treatment of Duchenne
muscular dystrophy (DMD)

Illuminate a potential
path forward