Cardiomyopathy is a disease of the heart muscle that can impair the heart’s ability to pump blood effectively and may lead to heart failure, arrhythmias, or sudden cardiac death.
Phase 1 data in 17 adults with Friedreich's ataxia cardiomyopathy showed AAVrh.10hFXN (LX2006) was generally well tolerated and showed signs of biological activity. The gene therapy is being advanced into a larger trial.
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Prothena reported Q4 2025 adjusted loss of 45 cents per share, meeting estimates, while ending the year with $308.4 million in cash. Roche and Novo Nordisk advanced partnered programs prasinezumab and coramitug into Phase III trials.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07591038 |
PET/CT Imaging in Carriers of TTR Mutations |
NOT_YET_RECRUITING | |
| NCT07574697 |
AIC Genotyping Study |
RECRUITING | |
| NCT07541833 |
Effectiveness and Treatment Patterns of Mavacamten in Patients With Obstructive Hypertrophic Cardiomyopathy in Japan (MANAGE-HCM) |
RECRUITING | |
| NCT07515235 |
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies |
RECRUITING | |
| NCT07479641 |
A Bioequivalence Study of Two Formulations of HRS-1893 Tablets in Healthy Participants |
RECRUITING | PHASE1 |
| NCT07428031 |
French Registry: Late Gadolinium Enhancement in Hypertrophic Cardiomyopathy |
NOT_YET_RECRUITING | |
| NCT07308704 |
A Study Of Deep Learning For Echo Analysis, Tracking, And Evaluation |
RECRUITING | |
| NCT07272330 |
A Phase I Trial on the Relative Bioavailability and Food Effect of HRS-1893 Tablets in Healthy Subjects |
COMPLETED | PHASE1 |
| NCT07154355 |
Community Exercise Treating Effect on Cardiopulmonary Disease Patients |
RECRUITING | NA |
| NCT07116525 |
A Study Assessing Arrhythmia Mapping With a Multi-Electrode Mapping Catheter |
RECRUITING | NA |
