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Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
Based on its novel mechanism of action, GSK’s gepotidacin could provide a new oral option for the treatment of gonorrhoea.
EU approval for Roche’s subcutaneous OCREVUS is anticipated mid-2024, depending on acceptance of regulatory submission.
Data from the pre-specified interim analysis of Fabhalta® (iptacopan) indicate a statistically significant reduction in proteinuria reduction.
In third line, MaaT013 represents the highest overall survival in acute graft-versus-host disease (aGvHD) when compared to reported literature evidence, according to MaaT Pharma.
Some patients had excellent responses in Phase II of an innovative trial of vosoritide in paediatric hypochondroplasia, data shows.
Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
Research suggests that for every five mothers given low dose esketamine, one major depressive episode could be prevented.
New data suggests the small molecule therapeutic could also benefit autoimmune disorders with involvement in the central nervous system, such as multiple sclerosis.
In the fourth instalment of EPR's ‘Microbiome therapeutics: microscope to medicine’ series, Emilie Plantamura, Deputy Chief Medical Officer at MaaT Pharma, examines the promising potential of microbiome therapeutics beyond Clostridium difficile infection, particularly in the onco-haematological field.
The novel combination cancer therapy facilitated “exceptional” responses in some patients enrolled in a Phase II study for endometrial cancer.
With no treatments approved for propionic acidaemia, Moderna’s LNP-encapsulated mRNA therapy could offer a new therapeutic option for patients, new data suggests.
The expanded authorisation of Reblozyl is based on a Phase III trial which showed that the treatment nearly doubled the percentage of certain transfusion-dependent anaemia patients achieving haemoglobin increase, compared to epoetin alfa.
In this Q&A, Jacqueline Barry, Chief Clinical Officer for Cell and Gene Therapy Catapult, elucidates on how the Advanced Therapy Treatment Centre (ATTC) Network can strengthen UK ATMP clinical trials.
A Phase I/IIa trial suggests that a subretinal gene therapy for wet age-related macular degeneration (wet AMD) could reduce the need for patients to receive anti-VEGF injections.
