Gene therapy for rare muscular dystrophy presents preliminary promise
Initial clinical results for a gene therapy being evaluated in limb-girdle muscular dystrophy has shown evidence of transgene expression in the three-month muscle biopsy.
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Initial clinical results for a gene therapy being evaluated in limb-girdle muscular dystrophy has shown evidence of transgene expression in the three-month muscle biopsy.
According to a report on policy development in the UK, the Future Clinical Trials Bill will mandate transparent trial registration and facilitate quicker regulatory approval.
The Clinical Trial Application sent to the MHRA is for a combination drug with the potential to become a cost-effective treatment against early Alzheimer’s disease.
A new, easy-to-use treatment option for advanced Parkinson’s could benefit around 900 adult patients on the NHS, according to National Institute for Health and Care Excellence (NICE) final draft guidance.
Dr Mark Rutstein, Senior Vice President, Head of Oncology Clinical Development at Daiichi Sankyo, highlights key data about the company’s antibody-drug conjugate (ADC) being investigated for breast and lung cancer, and shares insight into why ADCs could replace current standards of care in oncology.
The first targeted therapy for certain adults with the rare blood cancer myelodysplastic syndromes, has been approved by the US Food and Drug Administration (FDA).
Pharmaceutical quality standards are on the rise, putting pressure on development teams. See how Novartis is using Luminata to manage impurities data.
The Medicines and Healthcare products Regulatory Agency (MHRA), US Food and Drug Administration (FDA) and Health Canada have issued guidelines to reduce the regulatory burden for machine learning-enabled medical devices.
Progress, innovation and sustainability across the pharmaceutical supply chain were recognised at the 2023 CPHI Pharma Awards.
The first single vaccine to immunise against the five most common meningococcal disease serogroups in adolescents has been approved in the US.
With the pharmaceutical industry placing greater emphasis on sustainability, as a material, paper will play a key role in Europe’s packaging and labelling market between 2023-2028, research predicts.
Carrie Taylor, Director of Research & Development at BOC Sciences examines the key pathologies in Alzheimer’s and highlights several promising siRNA drug delivery systems that offer potential in helping to overcome the neurodegenerative disease.
Phase IIb study results show evidence of sustained, durable complete responses beyond one year with an intravesical gemcitabine delivery system for a high-risk bladder cancer.
Roche is set to gain rights to develop, manufacture and commercialise a novel antibody treatment with first-in-class and best-in-disease potential for inflammatory bowel disease (IBD), under a new acquisition agreement.
Strong evidence of long-term efficacy of the IL-13 inhibitor lebrikizumab for atopic dermatitis has been demonstrated in Eli Lilly’s two-year extension trial.