AVXS-101: Being developed for spinal muscular atrophy (SMA).
Brief information about it:
AVXS-101, now known as Spinraza, is a gene therapy medication used to treat spinal muscular atrophy (SMA), a rare genetic disorder that causes muscle weakness and wasting.
Uses:
Spinal muscular atrophy (SMA): Spinraza is approved to treat all types of SMA in infants, children, and adults.
Mechanism of action:
Spinraza is a spinal muscular atrophy (SMA) treatment that works by replacing the missing SMN protein in the body. This protein is essential for the survival of motor neurons, the nerve cells that control muscles.
Elimination half life:
The elimination half-life of Spinraza can vary depending on individual factors. It is generally excreted from the body within a few weeks.
Route of Administration:
Spinraza is administered as a series of intrathecal injections into the spinal fluid.
Side effects:
Spinraza can cause side effects, including:
- Back pain
- Headaches
- Fever
- Nausea and vomiting
- Respiratory problems
Dose:
The recommended dose of Spinraza varies depending on the patient’s age and weight. It is typically administered as a loading dose followed by maintenance doses.
Precautions:
· Consult a doctor: Before receiving Spinraza, consult with a healthcare professional to ensure it is safe for you and to discuss potential risks and benefits.
· Medical history: Inform your doctor about any personal or family history of conditions such as allergies, infections, or immune system disorders.
· Infections: Spinraza can weaken the immune system, making you more susceptible to infections.
