Action FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based
Recent advancements in stem cell technology open a new door for patients suffering from diseases and disorders that have yet to be treated. Stem cell-based therapy, including human pluripotent stem cells (hPSCs) and multipotent mesenchymal stem cells (MSCs), has recently emerged as a key player in regenerative medicine. hPSCs are defined as self-renewable cell types
Two children who received a Novartis gene therapy for their neuromuscular disease died following treatment, spotlighting its risks and renewing questions about the safety of genetic medicines like it. The patients developed acute liver failure between five and six weeks after infusion with the gene therapy, called Zolgensma and approved to treat spinal muscular atrophy,
Hannover Medical School doctors successfully treat three-year-old girl / Publication in “Nature Cardiovascular Research“ Clinical researchers at Hannover Medical School (MHH) have succeeded for the first time worldwide in stopping the usually fatal course of the disease in severe pulmonary hypertension thanks to a novel therapeutic approach. A three-year-old girl suffering from so-called pulmonary arterial
Given recent progress in cell therapy research, it is clear that the engineering disciplines outlined in this Review will play an increasing role in the creation of new product pipelines with improved safety, efficacy and accessibility for patients. Recent scientific advances have not only demonstrated the potential impact of technologiesdeveloped by each of these fields,
The research group led by Professor Misao Fujita conducted a study to estimate the total amount of refund the Japanese government would pay if a patient received unproven cell interventions and applied for a medical expense deduction. Some regenerative medicines whose safety and efficacy have not been fully confirmed in clinical trials and other studies are offered
Cedars-Sinai Investigators Found It Also Reduced Scarred Heart Tissue in Animals Vesicles secreted from human heart cells may repair damaged tissue and prevent lethal heart rhythm disorders, according to a new study from investigators in the Smidt Heart Institute at Cedars-Sinai. The research, published in the European Heart Journal, could lead to a new way to
Cell Therapy Developed by Smidt Heart Institute Leader Delays Disease Progression in Patients Who Have Few Treatment Options A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles–including the heart muscle–in Duchenne muscular dystrophy patients, a new study published in the international peer-reviewed journal The Lancet shows. If the HOPE-2 study’s
Natural killer (NK) cells derived from cord blood that were activated and complexed with a CD30/CD16A bispecific antibody elicited an 89 percent overall response rate in patients with relapsed or refractory CD30+ lymphoma, according to results from a phase I/II clinical trial presented at the AACR Annual Meeting 2022, held April 8-13. “We were favorably surprised by the
Experts say careful application of advanced tech could usher in a golden age of healthcare New technologies could enable medicine to progress in leaps and bounds, but only with the right regulatory and ethical frameworks. That was one of the messages from panelists discussing the future of medicine at the Science|Business conference Horizon Europe: The first assessment as
