Patient or Disabled Group
Entrada given go-ahead to test DMD therapy after two-year hold
Entrada, Muscular Dystrophy, Duchenne, DMD, United States Food and Drug Administration, Adult, Exons
Heartbeat Chronicles: Inspiring Stories and Breakthroughs in Cardiovascular Health
Heart disease, patient stories, medical advancements, cardiac research, heart health, cardiovascular breakthroughs
Eisai Anticipates Growth Acceleration for Alzheimer’s Drug Leqembi Despite Slow Initial Uptake
Leqembi, Alzheimer’s disease, Eisai, expansion phase, sales growth, diagnostics, maintenance dosing, patient adoption
FDA Urges Enhanced Authority to Combat Pediatric and Neonatal Medical Device Shortages
FDA, Medical Device Shortages, Pediatric Patients, Neonatal Patients, Supply Chain Vulnerabilities, Regulatory Authority
Novartis Achieves Pivotal Trial Success for Intrathecal Zolgensma in Older SMA Patients After FDA Delay
Novartis, Zolgensma, Intrathecal formulation, Spinal muscular atrophy (SMA), FDA approval, Pivotal phase 3 trial, Older SMA patients
Celebrating Visionaries: Insights from the 2024 Fierce 50 Honorees
Fierce 50, 2024 Honorees, Healthcare Innovation, Biopharma, Health Equity, Patient Advocacy
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.