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Compared to plasma-based therapies, the only recombinant ADAMTS13 protein in development showed strong efficacy in a trial for congenital thrombotic thrombocytopenic purpura (cTTP), Takeda revealed.
Strategies to further accelerate real-world evidence generation to aid EU regulatory decision-making are needed, states a European Medicines Agency (EMA) report.
A novel RNA CAR-T cell therapy demonstrated long-term clinical benefit for most generalised myasthenia gravis (gMG) patients in a landmark study.
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
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An FDA-approved anti-inflammatory drug can reduce cardiovascular disease risk by an additional 31 percent in adults, on top of standard of care, clinical data has shown.
At the 2023 ERA Congress, Chinook Therapeutics revealed Phase I data for its first-in-class oral small molecule LDHA inhibitor.
An osteoporosis study has demonstrated a high delivery success rate for a breakthrough technology that converts injections into an oral pill, data from ENDO 2023 showed.
A GSK-funded paper on pharmacovigilance stated that the pharma industry needs to harness the properties of data to allow optimal use of AI, otherwise "we will fail to do all we can for patient safety".
UCB Pharma's Chief Medical Officer Iris Loew-Friedrich, shares her perspective on digital innovation in clinical trials plus current challenges in clinical data management and how it could evolve in the future.
The US Food and Drug Administration (FDA) has approved the first bispecific antibody with a fixed-duration treatment in (R/R) diffuse large B-cell lymphoma (DLBCL).
If approved, VLA1553 could become the first licensed chikungunya vaccine available to address this unmet medical need.
CNS drug development is complex, and partnering with an integrated CRO/CDMO can reduce your timelines by up to 40%. Learn more in The Altascientist.
Researchers have revealed positive preliminary data from a CRISPR gene therapy trial for sickle cell disease.
Astrazeneca’s small molecule tyrosine kinase inhibitor reduced the risk of death by 51 percent in non-small cell lung cancer (NSCLC), a major Phase III study has shown.
