Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
The cardiovascular biologics market is projected to reach $4.23 billion by 2033, while the cell therapy market is expected to reach $5.38 billion by 2032, driven by RNA-based therapies and regulatory approvals.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07695701 |
Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis |
RECRUITING | PHASE4 |
| NCT07654855 |
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations |
NOT_YET_RECRUITING | |
| NCT07644325 |
Concordance for Transthyretin Amyloidosis Between Synovial Biopsy and Anterior Carpal Ligament Biopsy |
RECRUITING | NA |
| NCT07205666 |
The Eplontersen Pregnancy and Lactation Outcomes Study |
RECRUITING | |
| NCT06921408 |
Retrospective Identification of Scintigraphic Cardiac Amyloidosis (RISCA) |
NOT_YET_RECRUITING | |
| NCT06563895 |
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant |
RECRUITING | PHASE3 |
| NCT06465810 |
Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis |
RECRUITING | |
| NCT05758493 |
Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis |
RECRUITING | PHASE2 |
| NCT05023889 |
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy |
COMPLETED | EARLY_PHASE1 |
| NCT03588468 |
Expanding the Biomarkers in Familial Amyloid Neuropathy: MRI and Motor Unit Estimation by Electrophysiological Study |
COMPLETED | NA |
