Severe (severity modifier)
Gene Therapy Delivers Long-Term Immune Protection in Children With Rare Immune Disorders
gene therapy; long-term immune protection; ADA-SCID; severe combined immunodeficiency; LAD-I; leukocyte adhesion deficiency; rare disease; pediatric immunology; clinical trial; curative treatment
Ionis Builds Case for Wider Tryngolza Use in Severe Lipid Disorders Following Positive Phase 3 Results
Ionis Pharmaceuticals; Tryngolza; olezarsen; severe hypertriglyceridemia; lipid disorder; Phase 3 trial; familial chylomicronemia syndrome; acute pancreatitis; FDA; drug indication expansion
Sanofi Acquires China Rights to Arrowhead’s Plozasiran for $395 Million
Sanofi; Arrowhead Pharmaceuticals; Visirna Therapeutics; Plozasiran; China rights; RNA interference; Familial chylomicronemia syndrome; Severe hypertriglyceridemia; biotech partnership; milestone payments
CDC advisors broaden RSV vaccine recommendations to at-risk adults in their 50s
Adult, Respiratory syncytial virus, Centers for Disease Control and Prevention (U.S.), Vaccines, Recommendation, Age, Arexvy, Chronic Condition, Severe (severity modifier), 50–59, broaden
ACC25: Merck & Co.’s Winrevair reduces mortality in most severe PAH patients
Pulmonary arterial hypertension, Winrevair, Severe (severity modifier), Mortality Vital Statistics
Surrozen sets its sights on ophthalmology after binning liver programme
Ophthalmology specialty, Severe (severity modifier)
GSK’s Depemokimab Shows Promising Results in Phase 3 Trials, Reducing Asthma Attacks by Half
GSK, Depemokimab, Asthma, Phase 3 Trials, Long-Acting Biologic, IL-5 Inhibitor, Severe Asthma, Type 2 Inflammation
Almirall’s Ebglyss Gains NICE Recommendation for Atopic Dermatitis Treatment
Ebglyss, lebrikizumab, atopic dermatitis, NICE recommendation, Almirall, NHS England, biological therapy, moderate to severe atopic dermatitis
Vertex Announces Positive Results for VX-880 in Type 1 Diabetes Treatment
Vertex Pharmaceuticals, VX-880, Type 1 Diabetes, Stem Cell Therapy, Insulin Independence, Severe Hypoglycemic Events
FDA Grants Emergency Authorization for Invivyd’s Pemgarda Antibody Prophylactic Against COVID-19 in Immunocompromised Individuals
Invivyd, Pemgarda (pemivibart; formerly VYD222), Half-Life Extended Monoclonal Antibody, COVID-19 Prophylactic, Immunocompromised Patients, Emergency Use Authorization (EUA), FDA Approval, Moderate to Severe Immune Compromise, Pre-exposure Prophylaxis (Prevention) of COVID-19 in Adults/ Adolescents