DUCHENNE overview
What Is Duchenne?
Understanding Duchenne
What Is Duchenne?
Duchenne muscular dystrophy (Duchenne) is a rare genetic condition that causes muscles to become weaker over time. It affects muscles throughout the body, including those used for movement, breathing and heart function, and it is most often diagnosed in early childhood.
Duchenne
at a Glance
Affects ~1 in every
5,000 live male births
10,000–15,000 people in the U.S., representing significant unmet need1,2
Skeletal & cardiac muscle are affected early and continuously
Duchenne is caused by changes in the gene that provides instructions for making dystrophin, a protein that helps keep muscles strong and protected. Without enough functional dystrophin, muscle cells are more easily damaged, leading to increasing muscle weakness over time.
Clinical Trials
01
SGT-003: A Phase 1/2 clinical trial of an investigational gene therapy treatment for Duchenne
A multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne.
Active and recruiting
Location: U.S., Canada, Italy, and the U.K.
02
SGT-003: A Phase 3 clinical trial of an investigational gene therapy treatment for Duchenne
A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy of a single intravenous (IV) infusion of SGT-003 in pediatric ambulant male participants with Duchenne.
Active and recruiting
Location: Australia and Canada (with planned expansion into European countries and exploration of potential sites in the U.S., subject to receipt of regulatory approvals).
Patient Stories
Resources
Learn more about Duchenne through the following organizations:
References:
- Crossnohere NL, Armstrong N, Fischer R, Bridges JFP. Diagnostic experiences of Duchenne families and their preferences for newborn screening: a mixed-methods study. Am J Med Genet C Semin Med Genet. 2022;190(2):169-177. doi:10.1002/ajmg.c.31992
- Parent Project Muscular Dystrophy. About Duchenne Muscular Dystrophy. https://www.parentprojectmd.org/about-duchenne/.
| Stages of Duchenne | Age | Physical Manifestation |
|---|---|---|
| Early Phase | Ages of 2 to 7 | Delays in early developmental milestones. |
| Transitional Phase | Ages of 6 to 9 | Difficulty walking due to muscle weakness. |
| Loss of Ambulation | Ages of 10 to 14 | Use of power wheelchair on a regular basis. Activities involving the arms, legs or trunk will require assistance. |
| Adult Stage | Ages 15+ | Life-threatening heart and respiratory conditions become more prevalent. |