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Officials with the FDA issued a statement to clarify data accuracy issues with recently-approved gene therapy Zolgensma (onasemnogene abeparvovec-xioi, AveXis), a product intended to treat spinal muscular atrophy (SMA) in children under 2 years old.

Pexidartinib (Turalio, Daiichi Sankyo) is indicated for adult patients with symptomatic tenosynovial giant cell tumor associated with severe morbidity or functional limitations and not amenable to improvement with surgery.