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Novartis’ Vijoice® (alpelisib) is the first and only treatment approved for the treatment of the rare spectrum of conditions associated with PROS in certain patients.
A team of scientists has developed a spherical nucleic acid (SNA) vaccine, which has demonstrated high efficacy as a COVID-19 therapy, as well as an anti-cancer treatment in rodents.
Lentiviral gene therapy reduces infections and bleeding episodes in children with a severe immune disorder Wiskott–Aldrich syndrome (WAS), finds new study.
An artificial intelligence (AI)-based test was able to predict cancer patients’ response to drugs more accurately than is currently possible with genetic testing, finds proof-of-concept study.
New data shows that patients taking Aduhelm® (aducanumab-avwa) had significant reductions in tau and amyloid beta after nearly 2.5 years.
New data demonstrates the long-term safety and efficacy of Evrysdi® (risdiplam) in a broad population of people with spinal muscular atrophy (SMA).
The Phase IIa influenza-challenge study will enrol 123 healthy adults and support the development of INNA-051 as a pan-antiviral nasal spray.
Will mRNA vaccines one day protect against HIV? Three investigational mRNA vaccines, all designed to combat HIV, have entered Phase I testing.
Trodelvy® (sacituzumab govitecan-hziy) was found to significantly reduce the risk of disease progression or death in certain breast cancer patients.
Over 90 percent of β-thalassemia patients treated with Betibeglogene autotemcel (beti-cel) achieved transfusion independence lasting over a year.
Two-year data shows Pompe disease patients treated with Nexviazyme® (avalglucosidase alfa) have sustained improvements in respiratory function and mobility.
New draft guidance means thousands of obese Brits could benefit from Novo Nordisk’s Wegovy™ (semaglutide), a drug shown to improve weight loss.
The one-time gene therapy treatment for rare, neurodegenerative disease metachromatic leukodystrophy (MLD) is the most expensive drug ever evaluated by NICE.
Moderna and IAVI take steps towards HIV vaccination, with initiation of a trial dosing patients with HIV immunogens delivered by mRNA.
Newly published Network Meta-Analysis (NMA) from Janssen indirectly compares all published Phase III data for approved treatments for adults with active Psoriatic Arthritis (PsA).
