About this disease

It took a long time for someone to put all of my symptoms together, despite my family history of hATTR amyloidosis.

Lane, living with the polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTR) and taking TEGSEDI®

This case represents one patient’s experience. Individual results may vary.

What is the polyneuropathy of hereditary ATTR amyloidosis?

What happens in patients with hATTR amyloidosis?

There are 2 types of ATTR amyloidosis

  • Wild-type ATTR amyloidosis is caused by changes to a specific protein that may occur in some people as they age; it is not caused by a genetic mutation
  • Hereditary ATTR amyloidosis is caused by a genetic mutation and can be passed down through family members

The polyneuropathy of hereditary ATTR amyloidosis is caused by a change in the gene that makes a specific protein in your body

  • Your genes

    You have a change or mutation in the gene that makes a protein called transthyretin (trans-THIGH-ree-tin), or TTR

  • Your liver

    TTR is made in the liver. Your gene causes TTR proteins to form in an unusual shape

  • Protein clusters

    The unusual shape causes TTR proteins to clump together in clusters. These clusters are called amyloid fibrils

  • Amyloid buildup

    Amyloid fibrils build up in the body and damage tissues and organs

A parent who has hereditary ATTR amyloidosis has a 50% chance of passing the gene mutation on to their child

What is polyneuropathy?

Hereditary ATTR amyloidosis may cause polyneuropathy, the damage of multiple nerves

Polyneuropathy symptoms can include

  • Tingling, numbness, or pain in the hands and feet
  • Difficulty walking
  • Dizziness from low blood pressure
  • Sexual dysfunction
  • Vomiting and feeling sick
  • Diarrhea or constipation
  • Unintended weight loss
The disease may also affect other parts of the body

Eyes

  • Eye floaters
  • Glaucoma

Heart

  • Heart disease or heart failure
  • Irregular heartbeat

Kidney

  • Kidney failure

Wrists

  • Carpal tunnel syndrome

Not all patients with hATTR amyloidosis will experience all of these symptoms.

TEGSEDI is not approved for and has not been shown to be safe and effective for the treatment of cardiomyopathy in patients with hereditary ATTR amyloidosis.

I started having symptoms about 4 or 5 years ago; I noticed the neuropathy in my fingers and toes. It was manageable but did progress and become painful.

This case represents one person’s experience, individual results may vary

Mike, living with the polyneuropathy of hereditary ATTR amyloidosis and taking TEGSEDI

I have come to believe that my cardiac issues may have masked other issues that were probably brewing behind the scenes… because I began to develop polyneuropathy and GI issues.

This case represents one person’s experience, individual results may vary

Lane, living with the polyneuropathy of hereditary ATTR amyloidosis and taking TEGSEDI

Before I became symptomatic, I was very active. I used to love riding my bicycle, dancing, racing motorcycles—so it was devastating when I couldn’t do these things anymore due to my condition.

This case represents one person’s experience, individual results may vary

Chuck, living with the polyneuropathy of hereditary ATTR amyloidosis and taking TEGSEDI

The importance of understanding disease progression

Don’t wait until it gets worse

If left untreated, symptoms of hATTR can get worse over time


Peripheral sensorimotor neuropathy can occur as tingling, numbness, or pain.

Increasing nerve damage can make daily tasks – such as fastening buttons, turning a key, or walking – more difficult over time. With the polyneuropathy of hereditary ATTR amyloidosis, some everyday activities may become more challenging. As your disease progresses, these same simple daily tasks may become more difficult.

These symptoms can advance quickly, but there are treatment choices available. It is critical to receive appropriate treatment as soon as possible to help preserve nerve function.

It is important for you and your doctor to consider treatment as soon as possible after confirming your diagnosis

Genetics

hATTR amyloidosis is passed down through family members

A parent who carries the gene mutation for hATTR amyloidosis has a 50% chance of passing it on to his or her child

It is important for people with the polyneuropathy of hereditary ATTR amyloidosis to talk openly with their families about it.

Polyneuropathy genetic family tree chart

You may want to speak with a doctor together to learn about your family’s genes and consider genetic testing as a family

For more information about hereditary ATTR amyloidosis or genetic testing, call 1-866-AKCEATX or visit hattrcompass.com.

Important Safety Information
Indication

TEGSEDI is a medicine that treats the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis. TEGSEDI is for use in adults only.

Important Safety Information

TEGSEDI can cause serious side effects including:

Low platelet counts (thrombocytopenia): TEGSEDI may cause the number of platelets in your blood to be reduced. This is a common side effect of TEGSEDI. When your platelet count is too low, your body cannot form clots. You could have serious bleeding that could lead to death. Call your healthcare provider immediately if you have:

  • Unusual bruising or a rash of tiny reddish-purple spots, often on the lower legs
  • Bleeding from skin cuts that does not stop or oozes
  • Bleeding from your gums or nose
  • Blood in your urine or stools
  • Bleeding into the whites of your eyes
  • Sudden severe headaches or neck stiffness
  • Vomiting or coughing up blood
  • Abnormal or heavy periods (menstrual bleeding)

Kidney inflammation (glomerulonephritis): Your kidneys may stop working properly. Glomerulonephritis can lead to severe kidney damage and kidney failure that need dialysis. Call your healthcare provider immediately if you have:

  • Puffiness or swelling in your face, feet, or hands
  • New onset or worsening shortness of breath and coughing
  • Blood in your urine or brown urine
  • Foamy urine (proteinuria)
  • Passed less urine than usual

Because of the risk of serious bleeding caused by low platelet counts and because of the risk of kidney problems, TEGSEDI is available only through a restricted program called the TEGSEDI Risk Evaluation and Mitigation Strategy (REMS) Program. Talk to your healthcare provider about how to enroll in the TEGSEDI REMS Program.

Do not use TEGSEDI if you have:

  • A platelet count that is low
  • Had kidney inflammation (glomerulonephritis) caused by TEGSEDI
  • Had an allergic reaction to inotersen or any of the ingredients in TEGSEDI. See the end of the Medication Guide for a complete list of ingredients in TEGSEDI

Before you start TEGSEDI, tell your healthcare provider about all of your health issues, including if you:

  • Have or had bleeding problems
  • Have or had kidney problems
  • Have received a liver transplant
  • Are pregnant or plan to become pregnant. It is not known if TEGSEDI can harm your unborn baby
  • Are breastfeeding or plan to breastfeed. It is not known if TEGSEDI can pass into your breast milk or harm your baby. Talk with your healthcare provider about the best way to feed your baby while you are taking TEGSEDI

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take vitamin A or beta-carotene supplements, blood thinners (anticoagulants), or drugs that affect blood clotting.

Required monitoring

Your healthcare provider will test your blood and urine to check your platelet counts and kidney and liver function before you start TEGSEDI. While you are receiving TEGSEDI, you will be monitored closely for symptoms, which includes checking your platelet counts every week (or more frequently as needed), kidney function every 2 weeks, and liver function every 4 months. If your healthcare provider has you stop taking TEGSEDI, you will need to continue to get your blood and urine tested for 8 more weeks after treatment.

TEGSEDI may cause serious side effects, including:

Stroke. TEGSEDI may cause a stroke. One person taking TEGSEDI had a stroke, which occurred within 2 days after the first dose. Get emergency help immediately if you have symptoms of stroke, including sudden numbness or weakness, especially on one side of the body; severe headache or neck pain; confusion; problems with vision, speech, or balance; droopy eyelids.

Inflammatory and immune system problems. Some people taking TEGSEDI had serious inflammatory and immune system problems. Symptoms of inflammatory and immune system problems included unexpected change in walking, weakness and spasms in legs, back pain, weight loss, headache, vomiting, and problems with speech.

Liver Effects. TEGSEDI may cause liver problems. Your healthcare provider should do laboratory tests to check your liver before you start TEGSEDI and every 4 months while you are using it. Tell your healthcare provider if you have symptoms that your liver may not be working right, which could include unexpected nausea and vomiting, stomach pain, being not hungry, yellowing of the skin, or having dark urine.

Allergic reactions. TEGSEDI may cause serious allergic reactions. These allergic reactions often occur within 2 hours after injecting TEGSEDI. Get emergency help immediately if you have any symptoms of a serious allergic reaction, including joint pain, chills, redness on palms of hands, muscle pain, chest pain, flushing, tremor or jerking movements, flu-like symptoms, high blood pressure, or difficulty swallowing.

Eye problems (low vitamin A levels). Treatment with TEGSEDI will lower the vitamin A levels in your blood. Your healthcare provider will tell you how much supplemental vitamin A to take every day; only take the amount they tell you to take. Call your healthcare provider if you get eye problems, such as having difficulty seeing at night or in low-lit areas (night blindness).

The most common side effects of TEGSEDI include injection site reactions (such as redness or pain at the injection site), nausea, headache, tiredness, low platelet counts (thrombocytopenia), and fever. These are not all of the possible side effects of TEGSEDI. Talk to your healthcare provider about any side effects you may be experiencing.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please see Medication Guide and full Prescribing Information, including boxed WARNING.

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