Also known as: deflazacort
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
Priovant Therapeutics' brepocitinib demonstrated significant efficacy in the Phase 3 VALOR trial for dermatomyositis, meeting its primary endpoint with a 15.3-point greater improvement in Total Improvement Score versus placebo at Week 52. The treatment showed meaningful corticosteroid tapering and improvements across all nine key secondary endpoints, with benefits sustained through one year. Additional analyses revealed rapid itch reduction and improved skin-related quality of life in patients.
Five intra-articular gene therapy platforms for knee osteoarthritis are in human trials in the US, while India prepares regulatory guidelines for mRNA, gene and cell therapies to accelerate innovation.
The FDA cleared NexEos Bio's IND application to proceed with a Phase II/III trial of NTX-1024 ophthalmic solution for vernal keratoconjunctivitis, following positive results from a 15-patient study in Mexico.
Long-term data from the QUASAR extension study show TREMFYA® maintained clinical remission in over 80% of ulcerative colitis patients at Week 140, with more than half achieving endoscopic remission and no new safety concerns observed.
Ocular Therapeutix announced positive topline results from SOL-1, its Phase 3 superiority trial of AXPAXLI for wet age-related macular degeneration, meeting primary and durability endpoints with high statistical significance.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07611214 |
PRESERVE: LUPKYNIS in Combination With Belimumab, Obinutuzumab or Anifrolumab in Patients With Lupus Nephritis |
RECRUITING | PHASE4 |
| NCT07546097 |
Comparative Effectiveness of Upadacitinib vs Corticosteroids as First-Line Therapy for Acute Severe Ulcerative Colitis（UPFRONT） |
RECRUITING | PHASE4 |
| NCT07513142 |
PPP Versus Corticosteroid for Thigh Muscle Injuries |
ENROLLING_BY_INVITATION | PHASE4 |
| NCT07474714 |
Hyaluronic Acid Injection in Idiopathic Carpal Tunnel Syndrome; is it Effective as Local Corticosteroids |
NOT_YET_RECRUITING | PHASE3 |
| NCT07223580 |
A Study Of Corticosteroid On Postoperative Pain After Ureteroscopy For Urinary Calculi |
RECRUITING | PHASE4 |
| NCT07199192 |
Steroid Treatment for Severe Acute Respiratory Infection (STAR) Trial |
NOT_YET_RECRUITING | PHASE4 |
| NCT07098897 |
To Evaluate the Effect and Safety of Telitacicept and Standard Treatment for 6months in IgA Nephropathy |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT07039019 |
Study of the Feasibility and Efficacy of PrednisonE for Non-specifiC pleuriTis |
RECRUITING | EARLY_PHASE1 |
| NCT06952686 |
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants |
WITHDRAWN | PHASE3 |
| NCT06905860 |
Intra-articular Infiltration of Platelet-Rich Fibrin vs Corticosteroid in Mild to Moderate Knee Osteoarthritis |
NOT_YET_RECRUITING | PHASE4 |
