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Developing novel therapies for NASH
In this exclusive interview, Dr Hank Mansbach, Chief Medical Officer of 89bio discusses clinical development of FGF21 therapy to treat fatty liver disease.
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Clinical Trials
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First gene therapy recommended for AADC
NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.
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Could Dupixent be the first biologic to treat COPD?
Dupixent® is the first biologic to demonstrate significant improvement in lung function compared to placebo, a pivotal Phase III trial has shown.
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BioNTech to jointly develop novel antibody for cancers
ONC-392, an anti-CTLA-4 monoclonal antibody for solid tumours will be jointly developed by BioNTech and biopharma OncoC4.
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Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
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First FcRn blocker approved in UK for myasthenia gravis
VYVGART (efgartigimod alfa-fcab) has been approved for adults with generalised myasthenia gravis by UK Medicines and Healthcare products Regulatory Agency (MHRA).
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Dual cystic fibrosis modulator therapy shows efficacy in four-year study
Cystic fibrosis patients can safely take a dual combination of cystic fibrosis modulator drugs for four years, a study has shown.
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Novel small molecule drug facilitates leukaemia remission
Leukaemia patients went into remission when given a novel drug containing a small molecule that inhibits menin and MLL1 interaction, a study showed.
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Injectable peptide rapidly reduces intracranial pressure
A study for idiopathic intercranial hypertension is the first to show rapid and a major reduction in brain pressure and monthly headaches.
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New NHS cell and gene therapy centre opens
The new Clinical Biotechnology Centre (CBC) in Bristol will manufacture cell and gene therapies to help boost the UK life sciences sector.
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Could default eligibility improve clinical trial enrolment?
The number of potential clinical trial participants increased from over 51,000 to 1.7 million after researchers applied a new approach to improve eligibility.
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First nasal monoclonal antibody COVID-19 treatment shows potential
COVID-19 and multiple sclerosis patients experienced reduced inflammation when given the first nasal monoclonal antibody in a pilot trial.
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Natural allopregnanolone made orally bioavailable
Designed to treat anxiety and postpartum depression, a biotherapeutics company has made natural allopregnanolone orally bioavailable without permanent chemical modification.
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Leading in Pharma: a woman’s pathway
Here, to mark International Women’s Day, Dr Ekaterina Malievskaia from COMPASS Pathways reveals her path to working in mental health and the pharma industry.
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Oral PCSK9 inhibitor substantially reduces cholesterol, study finds
One of the first oral PCSK9 inhibitors tested in clinical trials significantly reduced low-density lipoprotein (LDL) cholesterol levels in patients with high cholesterol and/or heart disease.
