Fetus treated with risdiplam for rare genetic disorder, a medical first
HQ Team February 27, 2025: A study published in The New England Journal of Medicine (NEJM) reveals that administration of the oral drug.
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Drugs
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Medical
US regulator clears Mirum’s oral drug for reducing cholesterol deposits
Mirum Pharmaceuticals Inc.’s oral cholesterol drug for treating a rare metabolic disease has been approved by the Food and Drug Administration.
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Pharma
AstraZeneca’s drug to treat rare blood vessel disease gets EU nod
AstraZeneca Plc’s drug Fasenra got approval from the European Union regulator for treating adult patients with a rare inflammation of blood vessels.