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.

Sep 12, 2024 · 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.

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.

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 and ...

SPINRAZA (nusinersen) injection, for intrathecal use . Initial U.S. Approval: 2016 _____ INDICATIONS AND USAGE _____ and as clinically needed. SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients (1)

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.