Myasthenia gravis is an autoimmune neuromuscular junction disorder causing fluctuating skeletal muscle weakness and fatigability, often affecting ocular, bulbar, and limb muscles. Disease results from autoantibodies that impair neuromuscular transmission.
CAR-T therapy reaches 150 patients in Ireland and expands into autoimmune trials for lupus and MS. Kyverna's miv-cel nears FDA approval for stiff person syndrome, and new research links gene variants to CAR-T outcomes.
Vertex's Phase 3 RAINIER trial of povetacicept in IgA nephropathy met its primary endpoint with a 52% UPCR reduction. The FDA granted rolling review, and Vertex plans full BLA submission by end of March. Shares rose on the news and analyst upgrades.
Retrospective study analyzed longitudinal data from six immunotherapy-naive MuSK-MG patients over 2013-2024, finding significant positive correlation between MuSK antibody levels and disease severity as measured by MG-ADL scores. The correlation was specific to MuSK antibodies, with total IgG showing no independent relationship.
TG Therapeutics reported positive Phase 1 data for subcutaneous BRIUMVI in myasthenia gravis and started a Phase 2 trial; five-year ULTIMATE data in JAMA Neurology showed sustained MS efficacy.
A retrospective study found that myocarditis within the first month after immune checkpoint inhibitor initiation was linked to higher mortality risk. The analysis identified 4,635 cases in VigiBase.
Three FcRn inhibitors are approved for generalized myasthenia gravis, with a head-to-head trial of efgartigimod and nipocalimab underway. Emerging therapies including telitacicept, gefurulimab, and CAR-T cell therapy (KYV-101) show promise, while Immunovant's batoclimab failed TED trials.
BioNxt Solutions Inc. receives European Patent No. 4539857 for its proprietary sublingual cladribine oral thin film drug delivery technology, providing protection across up to 39 European Patent Convention states through at least June 14, 2043.
Amgen received FDA and European Commission approval for Uplizna to treat generalized myasthenia gravis in adults who are anti-AChR or anti-MuSK antibody positive, offering twice-yearly maintenance dosing after initial loading doses.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07761026 |
AXIS MG Patient Registry |
RECRUITING | |
| NCT07673744 |
A Study to Evaluate the Efficacy and Safety of Maintenance Ublituximab Following Induction With Efgartigimod Administration in Participants With Myasthenia Gravis (MG) |
RECRUITING | PHASE2 |
| NCT07653984 |
Cohort Study on Neuroimmune Diseases in the Reproductive Age |
RECRUITING | |
| NCT07649187 |
Depressive Tendency and Counseling Outcomes in Thymoma-Associated MG |
COMPLETED | NA |
| NCT07478172 |
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease |
RECRUITING | NA |
| NCT07411963 |
Evaluation of the Condition of Patients Receiving EARLY Ravulizumab and Admitted in ICU for gMG Crisis |
RECRUITING | |
| NCT07392528 |
Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/ BCMA（QT-019C） in Patients With r/ r Neurological Autoimmune Diseases |
NOT_YET_RECRUITING | EARLY_PHASE1 |
| NCT07333196 |
Tongji NADs Cohort |
NOT_YET_RECRUITING | |
| NCT07319689 |
Predictors of Myasthenia Gravis Outcome Following Thoracoscopic Thymectomy: Comparative Study Between Thymomatous and Non-Thymomatous Patients |
COMPLETED | |
| NCT07304154 |
A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases |
RECRUITING | PHASE1 |
