FDA Expands Casgevy for Children 2+ with Sickle Cell Disease
The FDA approved the first gene therapy for children as young as 2 years old with sickle cell disease or transfusion-dependent beta thalassemia.
The FDA approved the first gene therapy for children as young as 2 years old with sickle cell disease or transfusion-dependent beta thalassemia.
Research using lung-on-a-chip technology shows that mechanical stress from asthma attacks causes tissue damage independently of inflammation.
Testing via oral swabs identified hidden systemic inflammation in patients with primary ciliary dyskinesia even when they were not experiencing acute illness.
A study of over 1.1 million American adults with asthma found that Black, Hispanic, and Asian patients use ICS, LABA and LAMA inhalers less frequently than white patients.
AERO-007 demonstrated significant lung function improvements over 24 hours in patients with moderate-to-severe COPD.
A 28-year-old cystic fibrosis patient underwent a 36-hour quadruple organ transplant for both lungs, a liver, and a kidney from a single donor.
Phase 3 trial results show significant reductions in clinical worsening and acute exacerbations for patients with idiopathic pulmonary fibrosis (IPF).
New studies suggest that airway and breathing failures are the primary causes of death in childhood food anaphylaxis, requiring a shift in emergency management protocols.
Phase 3 clinical trial data shows improvements in walking distance and duration for patients with autoimmune pulmonary alveolar proteinosis treated with inhaled molgramostim.