Patient or Disabled Group
Mesoblast Secures FDA Approval for Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric Steroid-Refractory Acute Graft-Versus-Host Disease
Mesoblast, Ryoncil, FDA Approval, Mesenchymal Stromal Cell Therapy, Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGVHD), Pediatric Patients, Cell Therapy
Empowering Patients: The Key to Faster, More Impactful Research
Patient-centered care, Patient engagement, Personalized medicine, Clinical research, Healthcare outcomes, Patient empowerment
Neurogene’s Rett Syndrome Trial Halted After Patient Death Linked to High-Dose Gene Therapy
Neurogene, Rett syndrome, gene therapy, AAV, clinical trial, patient death, high-dose complications
Vertex’s CASGEVY Marks Historic Milestone with First Commercial Patient Infusions, Paving the Way for 2025 Launches
CASGEVY, Vertex Pharmaceuticals, gene therapy, sickle cell disease, beta thalassemia, CRISPR/Cas9, FDA approval, commercial patient infusions, 2025 launches.
Beam Therapeutics Reports Promising Early Results in Sickle Cell Disease Base Editing Therapy, Despite Conditioning-Related Fatality
Beam Therapeutics, Sickle Cell Disease, Base Editing, Gene Therapy, BEAM-101, ESCAPE, Conditioning, Patient Death
FDA Approves Avadel’s Lumryz for Pediatric Narcolepsy Patients
Avadel Pharmaceuticals, Lumryz, narcolepsy, pediatric patients, FDA approval, sodium oxybate, cataplexy, excessive daytime sleepiness (EDS)
FDA Halts Kezar’s Lupus Nephritis Trial Due to Safety Concerns Following Four Patient Deaths
FDA, Kezar Life Sciences, lupus nephritis, zetomipzomib, clinical hold, patient deaths, safety concerns
Kezar Life Sciences Suspends Enrollment and Dosing in Phase 2b PALIZADE Trial Due to Safety Concerns
Kezar Life Sciences, PALIZADE trial, zetomipzomib, lupus nephritis, safety concerns, patient deaths, clinical trial halt
Aixial Group Partners with Medidata to Enhance Clinical Trial Efficiency
Aixial Group, Medidata, clinical trial data, Single Source of Truth, data management, clinical research, patient data privacy, database lock.
Future-Proofing Healthcare: Inclusive and Protective Patient Strategies
future-proofing, inclusive healthcare, patient-centered care, digital patient experience, data linkage, clinical trials, health and safety programs