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Nusinersen, [7] marketed as Spinraza, [4] is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. [8] [4] In December 2016, it became the first approved drug used in treating this disorder.
Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) [6] [9] and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA). [6] Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier. [6] [5] [10]
In June 2015, the European Commission granted orphan designation to the drug. [19] However, in July 2019, the drug was removed from the Committee for Medicinal Products for Human Use (CHMP) accelerated assessment program. [20] In May 2019, onasemnogene abeparvovec received US FDA approval as a treatment for children under two years old. [14]
Novartis is working to expand use of the drug in older children, in part by seeking approval for a second version of the drug, administered by spinal injection, for children with less severe SMA.
Teaser: FDA approves Roche's (RHHBY) new SMA drug, Evrysdi (risdiplam). Skip to main content. 24/7 Help. For premium support please call: 800-290-4726 more ways to reach us ...
The FDA and the European Medicines Agency accepted to review supplemental marketing applications seeking approval for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA).
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. [3] [4] [5] It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. [6]
Biogen Inc should slash the price of its spinal muscular atrophy (SMA) drug, and the $4 million to $5 million Novartis has said its experimental gene therapy for the disease is worth is excessive ...
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