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Roche’s injectable C5 inhibitor PiaSky recommended by NICE for rare blood disorder PNH

Paroxysmal nocturnal haemoglobinuria affects approximately 1.59 per 100,000 people in the UK [...]

Johnson & Johnson’s Yuvanci granted EC approval to treat pulmonary arterial hypertension

Yuvanci is now the only single tablet combination therapy for PAH patients in Europe [...]

FDA approves IntraBio’s Aqneursa to treat Niemann-Pick disease type C patients

The rare lysosomal storage disorder affects approximately one in every 100,000 to 120,000 births [...]

‘Causes That Count, Health’ report – key issues affecting global health

The report focuses on the physical and mental health issues that matter most to people around the world [...]

MHRA approves Pharming’s Joenja as first drug for rare immune disease APDS

Up to 40 people in the UK are affected by activated phosphoinositide 3-kinase delta syndrome [...]

Ipsen’s Kayfanda granted EC approval to treat cholestatic pruritus in Alagille syndrome

The rare genetic disorder affects approximately three in every 100,000 births globally [...]

British Skin Foundation receives £10,000 donation from Circle Health Group

The healthcare provider’s previous donation is supporting research into early melanoma detection [...]

A social imperative: pharma’s essential role in online medical accuracy

We have a real opportunity, and arguably a responsibility, to stem the flow of misinformation [...]

European Commission approves Yuvanci for pulmonary arterial hypertension treatment

Janssen-Cilag therapy becomes the first single tablet combination therapy for PAH in europe [...]

Sheffield A&E to implement life-saving HIV and hepatitis testing

New government scheme to offer routine opt-out blood tests [...]

Oxford drug design secures funding to combat cystic fibrosis lung infections

£3m programme to accelerate new antimicrobial treatment [...]

Cancer Grand Challenges launches global initiative to take on toughest problems

International think tanks to shape future of cancer research funding [...]

AlgoTx completes pivotal phase 2 trial for ground-breaking CIPN treatment

International trial marks leap forward in chemotherapy-induced neuropathy care [...]

Oncologica to advance urine-based prostate cancer screening test

There is currently no national screening programme for the disease in the UK [...]

NHS Trust to evaluate AI software for prostate cancer diagnosis

It is hoped that the tool will improve the diagnostic pathway and free up radiologists’ time [...]

FlyPharma Europe, Vienna – October 2024 conference

The conference will return to Vienna AirportCity on 23 to 24 October 2024 [...]

Authenticx CPO Eric Prugh on the impact of AI technology in pharmacovigilance

Artificial intelligence has emerged as a potential solution to inconsistent reporting of adverse events, which to this day remains one of the main challenges in the field of pharmacovigilance. [...]

Microbiome startup Sequentia Biotech heralds “new era” in omics with €10 million round

The Barcelona-based startup Sequentia Biotech has raised â10 million ($11 million) in a Series A round to finance the development of its omics-based technology for use in biomedicine, agritech and food. [...]

Women in Science - engineering life’s mysteries: Joaquima Guix Salichs’ journey

Joaquima Guix Salichs is the director of engineering and extraction technology at Bioiberica, where she leads a team of scientists focused on advancing extraction methods and solving large-scale industrial challenges. [...]

AbbVie announces positive phase 3 results for Parkinson’s drug

Pfizer withdraws sickle cell disease drug Oxbryta amid safety concerns

The US big pharma company Pfizer has voluntarily withdrawn the oral sickle cell disease treatment Oxbryta from the market and halted clinical trials of the drug as data indicate a high risk of severe adverse events. [...]

FDA greenlights first treatment for Niemann-Pick disease type C

The small molecule drug arimoclomol (Miplyffa) has got the green light from the U.S. Food and Drug Administration (FDA) for the treatment of the rare disease Niemann-Pick disease type C (NPC), which until now has lacked FDA-approved therapies. [...]

Biohaven’s real-world trial shows positive results for spinocerebellar ataxia drug

The US biotechâs neurological drug candidate showed a reduction in disease progression of at least 50% and the company will now file with the FDA. [...]

Insilico Medicine claims phase 2 win for AI-designed drug

The Hong Kong headquartered company says its idiopathic pulmonary fibrosis drug performed well in a phase 2a trial and met its primary endpoint. [...]