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Roche’s aim to widen Evrysdi’s label nabs speedy FDA review in young spinal muscular atrophy patients

Now poised for a speedy trip through the FDA’s regulatory review, Roche’s oral challenger in spinal muscular atrophy (SMA) could soon pick up a nod …

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After its $7.6B buyout fell through, Sobi scores NICE backing for rare disease drug Aspaveli

After rare disease drugmaker Sobi reportedly had a buyout scuppered by pharma giant AstraZeneca, the company has picked up a win that stands to bolster …

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Researchers Develop Diagnostic for Rare Cancer-Generated Glycoproteins

Engineered chemical trap detects rare glycoproteins emitted as cancer first develops. Researchers at the Georgia Institute of Technology have designed a boronic acid derivative that …

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