The interim results of a single-arm trial in children with autosomal recessive deafness 9 (DFNB9) show the benefits of binaural administration of gene therapy: better hearing with no serious adverse effects.
AAV Therapy: A Successful One-Patient SPG50 Gene Therapy Trial
Stem Cell–Based Gene Editing Strategy Shows Promise in DMD
FDA Grants 2 Approvals to Delandistrogene Moxeparvovec for DMD
Rett Syndrome Gene Therapy Granted RMAT Designation
Five Children, 2 Hearing-Improved Ears Each: Successful DFNB9 Gene Therapy
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
Advances in Colon Cancer: Delivering Dual-mRNA With Lysate-Based Nanoparticles
Proof-of-Concept Study Highlights Potential of Gene-Based Therapy for Timothy Disease
Gene Therapy Offers Promise in Treating Inborn Errors of Immunity
Valoctocogene Roxaparvovec Dramatically Reduces Bleeding in Hemophilia A
New Strategy Shows Promise for CD7-Positive Leukemia/Lymphoma
Phase 3 Data Show Exa-Cel Prevents Vaso-Occlusive Crises for At Least 1 Year in SCD
AE Analysis Finds Low Rate of T-Cell Malignant Neoplasms in CAR T Recipients
Review Finds Anti-BCMA CAR T-Cell Therapy Effective in Patients With MM
Gene, Light Therapy Combo Shows Promise Against Prostate Cancer Cells in Proof-of-Concept Study
Gene Therapy Well Tolerated in Wet AMD, Shows Promise in Visual Acuity
FDA Approves Arsa-Cel for Metachromatic Leukodystrophy
Dr Ben Rome Scrutinizes the FDA’s Accelerated Approval Pathway for DMD Treatments, Calls for Policy Reform
Experts Issue Call for Reform to DMD Drug Pricing, Trial Approval Process
Addressing CAR T Challenges: Enhancing Collaboration for Patient Care
Optimizing CAR T for Patients With BCL and CNS Involvement
Identifying Patients at High Risk of Myeloid Neoplasms After CAR T: Implications for Care, Surveillance
When Choosing Between Bispecifics and CAR T, the Best Therapy May Be What Is Accessible
In Adolescents With Obesity, FMT Shows No Negative Impact on Horizontal Gene Transfer
Pre-, Postinfusion Protocol Recommendations for Delandistrogene Moxeparvovec Use in DMD
What We’re Reading: Sickle Cell Treatment Access; Patent Thicket Attacks; US Syphilis Cases Rise
What We’re Reading: Phase 3 Gene Therapy Trial; COVID Infection Surge; Patients Maintain Anti-Obesity Drug Weight Loss
Analysis Explores Gene Therapy’s Potential to Be Cost-Effective in SCD
Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada
Updated Data for Odronextamab Show Durable Responses in R/R DLBCL, 73% Complete Response in R/R Follicular Lymphoma
Sarepta Therapeutics Seeks to Expand Delandistrogene Moxeparvovec Indications
Descriptions of Physical Limitations, Symptoms of DMD From the Patient, Caregiver Perspective
Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy
ICYMI: AMCP Nexus Conference Highlights
Gene Therapy for Mucopolysaccharidoses Type IIIB Requires More Testing, Research
FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease