Zolgensma®: gene therapy for spinal muscular atrophy approved for use in the UK
The National Institute for Health and Care Excellence (NICE; UK) has published draft guidelines on the use of onasemnogene abeparvovec (Zolgensma®) for the treatment of spinal muscular atrophy (SMA) in the NHS in England and Wales.
Scientists develop new gene therapy strategy to delay aging
Cellular senescence, a state of permanent growth arrest, has emerged as a hallmark and fundamental driver of organismal aging. It is regulated by both genetic and epigenetic factors.
Peeking inside 'mini-brains' could boost understanding of the human brain
'Mini-brains' are pin-head sized collections of several different types of human brain cell. They are used as a tool, allowing scientists to learn about how the brain develops, study disease and test new medicines.
Novel BafA protein shown to stimulate angiogenesis
Scientists from Fujita Health University (Aichi, Japan) have discovered a pro-angiogenic autotransporter released by bacteria of the genus Bartonella, which they have named Bartonella angiogenic factor A (BafA).
New method to boost supply of life-saving stem cells
Researchers at the Center for Genomic Regulation (CRG) in Barcelona and Columbia University in New York City have identified a protein that is critical for the expansion of typically scarce, life-saving blood stem cells.
The evolving role of platelet-rich plasma (PRP) in plastic surgery
Platelet-rich plasma (PRP) treatment, which involves injecting a small amount of a patient's own blood to release various growth factors from platelets, continues to increase in popularity.
Novel CAR-T therapy sees success in treatment-resistant myeloma patients
Idecabtagene vicleucel (ide-cel), a novel CAR-T therapy, has produced positive results in a Phase II trial against multiple myeloma.
