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.

Find the latest Ionis Pharmaceuticals, Inc. (IONS) stock quote, history, news and other vital information to help you with your stock trading and investing.

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 ...