Author name: Leonardo Arias

Ionis-Otsuka offer ‘groundbreaking’ improvement in function and survival for genetic type of ALS

Ulefnersen, an RNA-targeted therapy being developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, improved measurements of functional impairment and survival in patients with a rare, genetic ALS subtype in a Phase 3 trial. The partners plan to approach the FDA with the results to determine potential pathways for approval.

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Novo’s turnaround plan, weight loss wins, Ultragenyx’s Sanfilippo approval, IPO blitz

Novo plans to hit $23B in revenue by 2035, has further reduced its workforce by 4,000 and is boosting manufacturing capacity for its Wegovy pill, but investors are unconvinced it will be enough to turn things around; Viking Therapeutics and Roche report positive results for their weight loss assets; Ultragenyx snags FDA approval for its

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