Cost, functioning, applications, mechanism, and additional information about Zolgensma
Zolgensma, a groundbreaking treatment for Spinal Muscular Atrophy (SMA), has been making waves in the medical community since its FDA approval in 2019. This gene therapy offers a promising solution for children under the age of 2, but it's important to understand its limitations and potential side effects.
The treatment, known as onasemnogene abeparvovec, works by replacing an abnormal SMN1 gene with a normal one, addressing the genetic cause of SMA and improving motor neuron function long-term. Clinical trials and reports show that Zolgensma produces sustained SMN protein production, leading to improved muscle function and survival. However, it's important to note that Zolgensma hasn't been studied as a treatment for advanced SMA, which may lead to conditions such as paralysis of the arms and legs or the need for a ventilator to breathe.
While early results are promising for infants and young children, longer-term data beyond several years are limited. Serious side effects from Zolgensma may include high levels of troponin I, thrombotic microangiopathy, allergic reactions, liver damage, low platelet levels, and seizures. If a child already has liver problems before receiving Zolgensma, they may have a higher risk of liver damage.
Common side effects of Zolgensma include nausea and vomiting. Vomiting can cause dehydration and an electrolyte imbalance, so it's recommended to give the child drinks that contain electrolytes, such as Pedialyte. If vomiting is severe or doesn't get better with time, talk with the child's doctor.
Zolgensma is a one-time intravenous infusion given to children less than 2 years old. However, it's worth noting that clinical studies haven't looked at how effective Zolgensma may be if a child receives more than one dose.
Other drugs, such as risdiplam (Evrysdi) and nusinersen (Spinraza), can also treat SMA, and some may be a better fit for a child than Zolgensma. Before receiving Zolgensma, the doctor will check a child's platelet level, and it's essential to monitor this before and periodically after treatment.
Zolgensma interacts with live vaccines, so during corticosteroid treatment, your child shouldn't get any live vaccines. Examples of live vaccines include chickenpox, measles, mumps, and rubella (MMR), and nasal flu spray (FluMist). Talk with your child's doctor about whether their vaccine schedule should be adjusted before they receive Zolgensma.
SMA is a rare condition that damages nerve cells in the brain and spinal cord, leading to muscle weakness and trouble with activities such as breathing, speaking, swallowing, and walking. The cost of Zolgensma can vary and may require prior authorization from insurance companies. Financial and insurance assistance is available through the OneGene Program offered by AveXis Inc., the manufacturer of Zolgensma.
While Zolgensma offers hope for many families affected by SMA, it's crucial to understand its limitations and potential side effects. Always consult with a healthcare professional for personalised advice and guidance.
- Despite Zolgensma's success in improving motor neuron function in children with Spinal Muscular Atrophy, its effects on advanced SMA cases, such as paralysis or the need for a ventilator, remain undetermined due to limited research in these areas.
- Pfizer, through their subsidiary AveXis Inc., offers the OneGene Program, which provides financial and insurance assistance for families who need the costly treatment Zolgensma for their children affected by Spinal Muscular Atrophy.
- It's essential for parents to be aware of Zolgensma's potential interactions with live vaccines, such as chickenpox, measles, mumps, rubella, and nasal flu spray, and discuss adjusting the vaccine schedule with their child's doctor before receiving the treatment to avoid any complications.
