1258984-36-9

Basic information

• Product Name: Nusinersen
• Synonyms: Nusinersen
• CAS NO: 1258984-36-9
• Molecular Weight: 0
• Mol File: 1258984-36-9.mol
• Article Data: 0

Chemical Properties

• CAS DataBase Reference: Nusinersen(CAS DataBase Reference)
• NIST Chemistry Reference: Nusinersen(1258984-36-9)
• EPA Substance Registry System: Nusinersen(1258984-36-9)

Safety Data

• Hazardous Substances Data: 1258984-36-9(Hazardous Substances Data)

Usage

General Description

Nusinersen, also known by its brand name Spinraza, is a chemical compound designed to treat spinal muscular atrophy (SMA), a rare genetic disorder that affects the motor neurons in the spinal cord. It works by targeting the genetic cause of SMA and increasing the production of survival motor neuron (SMN) protein, which is vital for motor neuron function. Nusinersen is administered directly into the spinal fluid through intrathecal injection, where it can reach the motor neurons affected by SMA. Clinical trials have shown that Nusinersen can improve motor function and slow the progression of SMA in both infants and older patients, making it the first FDA-approved treatment for SMA. Despite its high cost and the need for repeated dosing, Nusinersen has been a game-changer for many individuals and families affected by SMA.