Improvement
Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease
Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation
Bristol Myers’ schizophrenia drug Cobenfy stumbles as adjunctive treatment, denting blockbuster plan
Cobenfy, Schizophrenia, Antipsychotic Agents, Adjunctive Orthodontic Procedure, Myers, Statistical Significance, Improvement, Single Agent Therapy
New Uplizna data drop in myasthenia gravis, while clock ticks on IgG4 decision
Uplizna, Myasthenia Gravis, Effectiveness, Improvement
Mixed pivotal data for Pfizer, Arvinas’ protein degrader in breast cancer
Pfizer, Vepdegestrant, Clinical Trials, ESR1 gene, Statistical Significance, Improvement, Progression-Free Survival, Market
Boehringer Ingelheim’s Nerandomilast Succeeds in Second Phase III Lung Fibrosis Trial, Paving Way for FDA Approval
Nerandomilast, Boehringer Ingelheim, Phase III trial, FIBRONEER-ILD, progressive fibrosing interstitial lung diseases (PF-ILDs), lung function improvement, FDA approval, phosphodiesterase 4B (PDE4B) inhibitor
Narsoplimab Demonstrates Significant Survival Benefit in Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab, Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA), Lectin Pathway Inhibition, MASP-2 Inhibitor, Rare Disease Treatment, Overall Survival Improvement
Advancements in Hematology: Shaping the Future of Blood Disorder Diagnosis and Treatment
Hematology, Blood Disorders, Sickle Cell Disease, Gene Therapy, Clinical Guidelines, Quality Improvement
Biogen’s Higher Dose Spinraza Shows Enhanced Efficacy in Treating Spinal Muscular Atrophy
Biogen, Spinraza, Nusinersen, Spinal Muscular Atrophy (SMA), Higher Dose Regimen, Clinical Trial Results, DEVOTE Study, Motor Function Improvement, Neurodegeneration Reduction
Scholar Rock’s Apitegromab Shows Significant Improvement in Spinal Muscular Atrophy Treatment
Spinal Muscular Atrophy (SMA), Apitegromab, Phase 3 Clinical Trial, Motor Function Improvement, Myostatin Inhibition
AstraZeneca and Daiichi Sankyo’s TROP2 ADC Demonstrates Clinically Meaningful Survival Improvement in Phase 3 Lung Cancer Subgroup
AstraZeneca, Daiichi Sankyo, TROP2 ADC, Datopotamab Deruxtecan, Lung Cancer, Phase 3 Trial, Survival Improvement, Nonsquamous Non-Small Cell Lung Cancer