Helpful answers to frequently asked questions
Are there other people affected by the condition?
Although hereditary ATTR (hATTR) amyloidosis is a rare condition, you're not alone. There are roughly 50,000 individuals worldwide also affected. If you're ever feeling isolated, there are amyloidosis support groups and other organizations that you can connect with.
How do I talk to my doctor about hATTR amyloidosis?
Start by learning as much as you can about the condition. Having an understanding of hATTR amyloidosis can help in your discussion with your doctor. When you meet with your doctor, discuss all the symptoms you're experiencing. Depending on your doctor's experience, he or she may refer you to a specialist to help make a treatment plan or discuss next steps.
What is ONPATTRO® (patisiran) and how does it work?
In individuals with hATTR amyloidosis, a genetic mutation causes a protein called transthyretin (TTR) to misfold and build up into clusters (amyloid deposits) throughout the body, leading to symptoms of the disease.
ONPATTRO is a medicine that treats the polyneuropathy of hATTR amyloidosis in adults. It addresses the underlying cause of the disease by decreasing the production of TTR protein. This results in a reduction of amyloid deposits.
What are possible side effects of ONPATTRO?
The most frequently occurring side effects in patients treated with ONPATTRO were respiratory infections, such as colds, sinus infections, and nasal congestion, and infusion-related reactions. The most common infusion-related reactions were flushing of the skin, back pain, nausea, stomach pain, difficulty breathing, and headache. The majority of side effects were mild to moderate in severity, although some were considered severe. Infusion-related reactions decreased over time.
Be sure to talk to your doctor about side effects you may be experiencing.
How do I receive ONPATTRO medication?
How can Alnylam Assist® help me?
Once you and your doctor make the choice to begin treatment with ONPATTRO and submit a Start Form together, Alnylam Assist® can offer a range of services and resources to help you get started on treatment and provide you with ongoing support. Alnylam Assist® can also help explain insurance coverage for ONPATTRO and financial assistance options for eligible individuals.