Orsini, a leader in rare disease pharmacy solutions, announced today that it is a specialty pharmacy partner for ITVISMA® (onasemnogene abeparvovec-brve), an FDA-approved intrathecal gene therapy for ...

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Couple say screening for spinal muscular atrophy is crucial so children can get life-changing drugs.

Sara Ghanem and Samar Farag remember the exact moment they became friends, and their lives changed forever. With the constant sound of machines beeping in the background and the hustle and bustle of ...

William, 16, supports Ellie, 15, who has spinal muscular atrophy, alongside his volunteer work with the Scouts, and other jobs - including being the milkman for his village of Kirkby Malzeard, near ...

SCHAUMBURG, Ill., Nov. 26, 2025 /PRNewswire/ -- Cure SMA, the leading nonprofit organization dedicated to supporting those impacted by spinal muscular atrophy (SMA), welcomes the FDA approval of ...

The FDA has approved Itvisma for the treatment of children two years and older, teens and adults living with spinal muscular ...

In today’s Pharma Pulse, the federal government prepares to unveil newly negotiated Medicare prices for 15 high-cost drugs, while Novartis receives FDA approval for Itvisma, a one-time gene therapy ...

Now, it’s worth noting Stock Advisor’s total average return is 1,002% — a market-crushing outperformance compared to 190 % ...

The U.S. Food and Drug Administration has approved Novartis' gene therapy for patients with a rare muscle disorder, the drugmaker said on Monday.

Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...

I used to have a crate here for JC. For the first two months [after his death], I would carry this pillow and stay in the ...