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“Landmark” genetic blindness CRISPR trial outcomes released
A Phase I/II trial has shown that a CRISPR-based gene editing therapy can be safely delivered to the retina and provide clinically meaningful outcomes.
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NICE recommends new haemolytic anaemia treatment
The National Institute For Health and Care Excellence (NICE)'s decision follows a reduction in the price of Pfizer’s small molecule treatment for sickle cell disease.
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MHRA publishes strategy on AI regulation
Incorporating artificial intelligence (AI) into the MHRA’s regulatory processes could help the agency focus on key priorities such as innovation.
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Long-acting antibody demonstrates promising efficacy in infant RSV
New real-world evidence from a long-term follow up study adds to evidence of the high efficacy of Beyfortus in infant RSV.
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CHMP meeting highlights: April 2024
In its April meeting, the EMA’s human medicines committee recommended eight new medicines, including treatments for cancer and autoimmune diseases.
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Accelerating spectroscopic analysis for data acquisition
A study has reported the first experimental evidence of time-domain compressed sensing, which could accelerate data acquisition in ultrafast spectroscopy.
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Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
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Rapid method for whole-genome fungal ID
The novel technique for whole-genome analysis of fungal species is “highly suitable” for quality and safety monitoring of medicinal materials, the paper suggests.
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Benefits of 3D printing for personalised medicine formulation
A study has demonstrated how 3D printing can be used to manufacture tailored, safe, and effective treatments for patients with rare diseases via pharmaceutical compounding.
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Research calls for greater investment in Alzheimer’s clinical trials
Despite some challenges, for the Alzheimer’s drug development pipeline, 2024 is a “learning year”, an expert on the disease says.
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Generics propelling pharmaceutical parenteral packaging market
Preference for small molecule prefilled syringes and a greater demand for prefilled insulin syringes for diabetes is driving growth of the pharmaceutical parenteral packaging market, a report states.
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Microbe-carrying particle recovery efficacy with manual surface sampling
Three different contact plate methods were tested to determine a convenient manual surface sampling technique for microbial contamination recovery, the paper explained.
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NICE recommends Novartis precision brain cancer treatment
An “innovative, life-extending treatment” for BRAF V600E mutation-positive glioma has been recommended for certain young people on the NHS.
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European Commission approves novel antibiotic
Authorisation of the antibiotic combination, which targets multidrug-resistant bacteria, could help to address the global challenge of antimicrobial resistance (AMR).
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Utilising rapid UHPLC for tablet QC analysis
The proposed UHPLC method is suitable for routine quantitative analysis of teneligliptin and pioglitazone, a paper reports.
