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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]
This list of over 500 monoclonal antibodies includes approved and investigational drugs as well as drugs that have been withdrawn from market; consequently, the column Use does not necessarily indicate clinical usage.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.
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.
The weight loss drug Zepbound, generically known as tirzepatide, is also now an approved medication to treat obstructive sleep apnea, per a Food and Drug Administration Dec. 20 press release.
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 ...
Misoprostol is approved for medical abortion regimens when administered at the office, but clinicians often give abortion patients the drug to be taken at home. [15] Modafinil is a central nervous system (CNS) stimulant medication used to treat sleepiness due to narcolepsy, shift work sleep disorder, and obstructive sleep apnea. It is often ...
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]
The U.S. Food and Drug Administration (FDA) granted onasemnogene abeparvovec-xioi various designations including fast track, breakthrough therapy, priority review, and orphan drug designations. [14] Additionally, the FDA awarded the manufacturer a rare pediatric disease priority review voucher and approved onasemnogene abeparvovec for AveXis ...
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