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NICE backs first disease-modifying drug for ARG1 deficiency

Immedica's Loargys is poised to become the first NHS therapy for ultra-rare genetic disorder ARG1 deficiency that can alter the course of the disease.

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NHS study will pick apart mental illness with data science

Volunteers with schizophrenia, bipolar, and depression will take part in an NHS study that will use genetic and health data to find new therapies.

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Report says Jim O'Neill's time at HHS is nearing its end

HHS Deputy Secretary Jim O'Neill – who is also head of the CDC – is said to be on the way out after a reshuffle of department leadership by RFK Jr.

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Bankruptcy threat looms as Quince runs out of options

Investor panic has poleaxed Quince Therapeutics share price, after a regulatory filing revealed the biotech's cash is running out.

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PTC pulls file for Duchenne therapy on FDA feedback

PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

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BridgeBio gains on phase 3 dwarfism data

Shares in BridgeBio rose after it revealed data from a trial of infigratinib in achondroplasia, raising the prospect of the first oral treatment.

NICE backs first disease-modifying drug for ARG1 deficiency

NHS study will pick apart mental illness with data science

Report says Jim O'Neill's time at HHS is nearing its end

Bankruptcy threat looms as Quince runs out of options

PTC pulls file for Duchenne therapy on FDA feedback

BridgeBio gains on phase 3 dwarfism data

ASCO 2026: All about DNA damage response, with Luke Piggot

Discover a new era of healthcare panel engagement: Grounded ...

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