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Novartis: wins Japan approval for SMA drug

(CercleFinance.com) - Swiss drugmaker Novartis said on Thursday that it has won Japanese approval for a new treatment of spinal muscular atrophy (SMA) in patients under the age of two.


A rare neuromuscular disease caused by a lack of a functional gene, SMA results in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movements.

Novartis has also unveiled the results of three pivotal Phase III clinical trials for inclisiran, an investigational drug for hyperlipidemia in adults, showing that after two starter doses, twice-yearly subcutaneous dosing resulted in durable and potent cholesterol reductions versus placebo.

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