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.

Nusinersen, marketed in the U.S. as Spinraza ® (Biogen) is the first therapy approved for the treatment of SMA. SMA results from mutations in a gene known as SMN1, which encodes a protein (Survival Motor Neuron, or SMN) important for motor neuron survival.

SPINRAZA ® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. Increased risk of bleeding complications has been observed after administration of similar medicines.

2024年9月12日 · What is nusinersen? Nusinersen works by helping the body produce a protein essential to the function of nerves that control muscle movement. This protein is lacking in people with an inherited disorder called spinal muscular atrophy caused by a mutation in a specific gene.

FDA approved Spinraza (nusinersen), the first drug approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength...

It is a novel modified antisense oligonucleotide designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron protein deficiency. Nusinersen has been studied for safety, pharmacokinetics, and efficacy in …

SPINRAZA ® (nusinersen) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.

2023年4月4日 · Spinraza (nusinersen) is a type of medication called an antisense oligonucleotide. It is used to treat children and adults with spinal muscular atrophy (SMA), a type of motor neuron disease.

SPINRAZA ® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients. Increased risk of bleeding complications has been observed after administration of similar medicines.

Nusinersen is a small piece of man-made genetic material (a synthetic antisense oligonucleotide). It targets the SMN2 gene and enables it to produce more functional, full-length SMN protein. Nusinersen is manufactured by the pharmaceutical company Biogen …