MedPage Today3d
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
2minutemedicine.com5d
Beti-cel gene therapy achieves high rates of transfusion independence in severe β-thalassemia
There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron ...
PCGamesN3d
Elden Ring Nightreign beta start date and end times
When is the Elden Ring Nightreign beta? The Lands Between beckons us once more for a standalone co-op adventure that introduces a roguelike loop into FromSoftware’s formula. Playtests are the ...
SciELO6d
Pseudogaucher cells in a patient with α-thalassemia minor and S-hemoglobin carrier
The presence of PGC has been documented in different hematological diseases being most common chronic myeloid leukemia but also in myelodysplastic syndromes, myeloma and other lymphoproliferatives, ...
Nature5d
Structure-based drug design articles from across Nature Portfolio
Structure-based drug design is the design and optimization of a chemical structure with the goal of identifying a compound suitable for clinical testing — a drug candidate. It is based on ...
The Scientist5d
The Scientist - Home
Neurons that secrete the neuropeptide orexin help mice decide between exercise and snacking.