A new Italian White Paper reveals women with rare diseases wait two years longer than men for a diagnosis, while Georgia expands state-funded care for rare neuromuscular conditions to adults. The report also highlights regional disparities and economic burdens on patients and carers.
Servier will acquire Edgewise Therapeutics' muscular dystrophy business, including the drug sevasemten, for up to $2.65 billion. Sevasemten recently faced an FDA rejection for accelerated approval in Becker muscular dystrophy but is advancing to a Phase 3 trial in 2026. The drug has received multiple FDA and EMA designations for both Becker and Duchenne muscular dystrophy.
Edgewise Therapeutics and IDEAYA Biosciences released annual financial reports detailing clinical trial progress and strategic initiatives. Both companies are advancing multiple clinical-stage programs while managing significant operating expenses.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07740057 |
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function |
NOT_YET_RECRUITING | |
| NCT07674758 |
Modeling Mortality in Duchenne Muscular Dystrophy Cardiomyopathy: Identification of Surrogate Outcome Measures for DMD Drug Trials |
RECRUITING | |
| NCT07378553 |
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies |
RECRUITING | |
| NCT06066580 |
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT05291091 |
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05257473 |
Defining Endpoints in Becker Muscular Dystrophy |
ACTIVE_NOT_RECRUITING | |
| NCT04972604 |
CureDuchenne Link®: A Resource for Research |
COMPLETED |
