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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]
AveXis, and later Novartis, spent less than $12 million up to the point of the drug’s approval — surprisingly little — to prove the therapy was safe and effective, the group estimated, based ...
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 ...
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 ...
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]
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage. See the list of FDA-approved therapeutic monoclonal antibodies in the monoclonal antibody therapy page.
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