What is spinraza and how it works?
Spinraza (nusinersen) has been approved by the FDA and is now available for treatment of patients with SMA due to SMN1 mutations. The CUIMC - SMA Clinical Research Center was the first site in the world to administer Spinraza in a clinical trial. Patients who meet the following criteria may be eligible for treatment with Spinraza:
Patients who have genetically confirmed SMA and meet the medical criteria for treatment.
Patients who are registered and receiving care with a ColumbiaDoctors provider.
Patients who are able to follow-up with their ColumbiaDoctors provider after treatment.
Patients who can be evaluated by their ColumbiaDoctors provider, including physicians and nurse practioners, six months after receiving their Spinraza treatment.
@natasha Spinraza is a brand-name prescription drug. It’s FDA-approved to treat spinal muscular atrophy (SMA) in adults and children. SMA is a rare genetic disorder* that affects the central nervous system, which includes your spinal cord and brain. Specifically, SMA is a type of motor neuron disease. This means it results from problems with the neurons (nerve cells) that control muscle movement. Over time, SMA leads to muscle weakness and trouble walking, speaking, breathing, and swallowing. 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 correctly