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More Hong Kong patients suffering from spinal muscular atrophy (SMA) will be able to enjoy a government subsidy to control ...
Biogen has been trying to build up its R&D pipeline as it faces increased competition for its biggest-selling products like ...
Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) is one of the 11 Best Mid-cap Healthcare Stocks to Buy According to Hedge Funds.
While leveraging the same mechanism of action as SPINRAZA® (nusinersen), but designed to achieve greater potency, salanersen can achieve high efficacy and enable once-yearly dosing.
Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease SMA.
In July 2025, Novartis Pharmaceuticals conducted a Phase III multi-center, single dose (1.2 x 1014 vector genomes), ...
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Spinal Muscular Atrophy Pipeline Therapies- OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, vesemnogene lantuparvovec, Olesoxime, NMD670 and others.
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Background Spinal muscular atrophy (SMA) Type 1 is a neuromuscular disorder that has traditionally resulted in death from respiratory failure before 2 years of age. Treatment with Nusinersen, an ...