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.

One of the latest breakthroughs in care for patients with spinal muscular atrophy is the FDA approval of a new medicine called nusinersen (brand name Spinraza) for the treatment of SMA in children and adults.

Sep 12, 2024 · 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.

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 …

Apr 4, 2023 · 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.

SPINRAZA contains nusinersen, which is a modified antisense oligonucleotide, where the 2’ hydroxy groups of the ribofuranosyl rings are replaced with 2’-O-2-methoxyethyl groups and the phosphate...

Nusinersen demonstrated a favorable long-term benefit-risk profile in this broad population of individuals with infantile- or later-onset SMA.