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Gene therapy trial facilitates major vision improvement
Some of the rare disease participants experienced fast and sustained improvement in their eyesight during the clinical trial, data shows.
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Drug Safety
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First-in-class combination therapy authorised for endometriosis
The three-in-one oral daily tablet provides an additional treatment option to help women in the UK manage their endometriosis-associated pain.
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Innovative hypertension polypill study achieves “significant” outcomes
The triple pill demonstrated “impressive” blood pressure control for the participants compared to standard of care, according to the study data.
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Mpox update – August 2024
This article discusses the impact of the recent mpox emergency following the outbreak of the virus in 2022, highlights the industry’s response, potential vaccine candidates and key recommendations for international management of the disease.
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European Commission licenses first treatment for acanthamoeba keratitis
Following its European approval, the small molecule treatment is set to become the standard of care for patients with the ultra-rare disease.
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First-of-a-kind trial holds promise for obesity and pre-diabetes treatment
The treatment reduces the risk of progression to type 2 diabetes and supports long-term maintenance of weight loss in adults with pre-diabetes and obesity or who are overweight, data indicates.
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Targeted therapies for chronic lymphocytic leukaemia in an evolving treatment landscape
Dr Mehrdad Mobasher, Chief Medical Officer for Hematology, BeiGene, discusses the evolution of therapies for chronic lymphocytic leukaemia (CLL), the promise of targeted treatments and what could be on the horizon of the therapeutic landscape for this disease.
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Innovative vaccine demonstrates sustained malaria immunisation in pregnancy
Follow up research to the presented findings could provide improved opportunities for preventing malaria in women during pregnancy.
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First-in-class therapy approved for chronic skin disease
The biologic treatment targets a key driver of prurigo nodularis, with trial data demonstrating improved itch as well as skin nodules for patients with the condition.
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Sustainable QC method for antimicrobial quantification
The new, cost-effective fluorimetric approach could help to mitigate clinical demand for the treatment of multidrug-resistant (MDR) bacterial infections.
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HDFN drug trial shows capability for alloimmune diseases
Based on the new data, nipocalimab could change the standard of care for treating the rare blood disease haemolytic disease of the foetus and newborn (HDFN).
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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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Personalised mRNA cancer therapy shown to boost immune response
The individualised neoantigen therapy could enable a precise, durable immune response, based on study findings in melanoma and lung cancers.
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FDA approves innovative engineered cell therapy
The accelerated approval by the US Food and Drug Administration (FDA) authorises the first engineered cell therapy for a solid tumour in the US.
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Alzheimer’s drug development roundup – July 2024
From exciting data on cell therapy and monoclonal antibodies, through to new regulatory developments, this article summarises some of the key developments within the Alzheimer’s therapeutic landscape over July.
