Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene abeparvovec, an investigational gene therapy.
Standardized Measurements Needed for AAVs in Gene Therapy, Study Finds
Gene Therapy Demonstrates High Efficacy in Severe β-Thalassemia
ICYMI: Highlights From AMCP Nexus 2024
New Evidence Highlights Stability of Lentiviral Gene Therapies
Phase 3 STEER Study of SMA Gene Therapy Meets Primary End Point
Dr Migvis Monduy on the Most Promising Areas of DMD Research
Dr Landon Marshall Discusses Gene Therapy Forecasting Benefits, Future Advancements
Payers, Providers Still Navigating Uncharted Waters for Gene Therapies
ICYMI: Highlights From AMCP Annual 2024
FDA Pauses PepGen’s DMD Drug Trial in the US
Top 5 Most-Read Gene Therapy Articles of 2024
Comparing CAR T-Cell Therapies for LBCL: Insights From CART-SIE Study
Ultra-High-Cost Gene Therapies Set to Influence Major Pharmacy Shifts
Gene Therapy Enhances Visual Processing for Inherited Retinal Disease
Expanding Access to Gene Therapies: Addressing Patient Burden, High Costs
Real-World Data Demonstrate Efficacy of Beti-Cel Gene Therapy
Viltolarsen Slows DMD Progression in Small Study
Sarepta Halts Development, Testing of DMD Therapy SRP-5051
FDA Approves Gene Therapy to Treat AADC Deficiency
Fidanacogene Elaparvovec Shows Strong Results in Open-Label Phase 3 Study
Medicaid Aiming to Improve Patient Access to High-Cost Therapies
A Year of DMD Gene Therapy Trial Failures
Different Cost-Effectiveness Models of CAR T Yield Similar Results
CNS-Directed AAV Gene Therapy: Immune Dynamics and Clinical Implications
Voretigene Neparvovec Helps Patients With RPE65-Related Retinopathy Regain Bilateral Function
Protein's Role in Insulin Signaling Could Have Implications for Gene Therapy
Meta-Study Analyzes Immunosuppressive Protocols for AAV Gene Therapy for Monogenic Disorders
Emerging Technology Engineers Oncogenic Mutation Into Cell Lines
Review: CAR T Cells Could Transform Pediatric SLE Therapy
Neurological Complications in CAR T-Cell Therapy: A Case of Parkinsonism
Looking to CAR T-Cell Therapy in Refractory Pediatric SLE
An Updated Snapshot of Gene Therapy Clinical Trials Worldwide
Novel CAR-E Platform Enhances CAR T-Cell Functionality in Cancer Treatment
High Costs of Sickle Cell Therapies Highlight Paradox of Drug Development
CRISPR/Cas9 Gene Therapy Holds Promise to Treat Epidermolysis Bullosa
Progress in Precise Gene Expression Control With CRISPR-Cas12f
Hemophilia A Gene Therapy Superior to Standard of Care in Phase 3 Trial
Key Considerations for Optimizing B-VEC in Dystrophic Epidermolysis Bullosa
