Merck rare tumour drug Ezmekly backed for NHS use
Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.
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Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.
Ionis' Zanvastro has been approved by the FDA for Alexander disease, becoming the first disease-modifying therapy for the devastating rare disease.
In a handful of strongly-worded warning letters, the FDA is cracking down on sales of unapproved peptides - even if they're labelled for research.
Federal funding from ARPA-H of $125m, awarded to five groups, is part of a drive to accelerate patient access to personalised genetic medicines.
Enhertu plus Perjeta has become the first new frontline therapy approved for advanced HER2-positive breast cancer in the EU for over a decade.
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