DUCHENNE overview
What Is Duchenne?
Understanding Duchenne
What Is Duchenne?
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
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:
The organizations listed are independent patient advocacy groups. Inclusion does not imply endorsement. This information is for awareness purposes only.
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. |