All Dr Matthew Klein articles
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News
Broad label FDA approval for phenylketonuria drug
Alongside Phase III data demonstrating significant efficacy in the rare disease, the FDA’s decision could position the small molecule as a future standard of care.
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NewsNovartis to advance novel Huntington's disease therapy
As part of the agreement with Novartis Pharmaceuticals, PTC will receive an initial $1 billion to develop the potential first-in-class oral therapy.