Video answer: Sma drug spinraza approved by fda
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SPINRAZA is an antisense oligonucleotide (ASO) designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
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Spinraza is a medicine used to treat 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. The disease is linked to a defect on chromosome 5q and symptoms usually start shortly after birth.
Spinraza is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Spinraza Dosage and Administration Dosing Information. Spinraza is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. Recommended Dosage
Spinraza. Generic name: Nusinersen (nue si NER sen) Brand name: Spinraza. Drug class: Miscellaneous uncategorized agents. Medically reviewed by Drugs.com. Last updated on Jan 6, 2021. Uses.
Spinraza contains the active drug nusinersen. It belongs to a class of drugs* called antisense oligonucleotides. Spinraza works by targeting a certain protein that helps nerve cells function...
Brand name: Spinraza Drug class: Miscellaneous uncategorized agents. Medically reviewed by Drugs.com on Sep 22, 2020. Written by Cerner Multum. Uses; Warnings; Dosage; What to avoid; Side effects; Interactions; FAQ
DESCRIPTION. Survival motor neuron-2 (SMN2)-directed antisense oligonucleotide. Used for adult and pediatric patients with spinal muscular atrophy (SMA); SMA is a rare and often fatal hereditary genetic disease affecting muscle strength and movement. Requires intrathecal dosing.
Spinraza. Spinraza® is the trade name of a medication that contains an active substance called nusinersen, a chemical belonging to the class of antisense nucleotides (ASO). ASO-s are sequences of nucleic acids mimicking human DNA. Nusinersen has been designed to attach to the SMN2 gene and modify its structure.
Nusinersen. Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder. Nusinersen has orphan drug designation in the United States and the European Union.
Learn about SPINRAZA, an FDA-approved pediatric & adult treatment from Biogen, including where to find treatment centers. See trial results and Safety info.