WAINUA
Patient Voices

Brigitte:

Hi, I am Brigitte. I'm 44 years old. I'm a mom of 2 teenage daughters.

I'm a certified personal trainer and also a group fitness instructor. I have what is officially known as polyneuropathy caused by hereditary ATTR amyloidosis or hATTR-PN.

Steve:

My name’s Steve, I'm Brigitte's husband. We've been married 20 years this year. Family time is very important to both of us.

Brigitte:

Basketball games, volleyball games, walks with the dog. We are on our ATV in the woods, all 4 of us. I first started feeling some of these symptoms when I was around 28 years old. I would have burning through my fingers, up and through my wrists, my lower extremities. I would have burning from my knees to my toes and at times it would wake me up at night. I could feel my heart racing and I'd say, "I can't breathe. I feel like there's an elephant standing on my chest. Something's wrong." I would take my blood pressure, 180 over 104. For well over 10 years I was misdiagnosed, no one would listen. I was diagnosed with POTS, with the postural orthostatic tachycardic syndrome. I was told because of my Crohn's diagnosis that I had peripheral neuropathy. I was also told that I had rheumatoid arthritis.

Steve:

Diagnosis after diagnosis that would turn out to not be the correct one.

Brigitte:

Talking to the medical professionals and not getting answers and not having someone believe me was horrific. It's like you're told, "You're anxious, it's all in your head, there's nothing wrong with you, you're fine." My grandfather had a lot of health issues similar to what I had. He was losing feeling in his feet and he was losing the function in his hands. He could not hold my children anymore and they finally decided to test him when it came back positive for hATTR-PN amyloidosis, which is hereditary.

My mother had to be tested and when she had a positive result, my brother and I had to be tested. My brother was negative, I was positive. I remember walking in the kitchen and my husband waiting with open arms saying, "We'll do anything. We will fly wherever you need to go. We will find you treatment. We will find you hope and help." Going through all the motions and all the different doctors until I found my doctor who said, "Yes, you need treatment. Yes, we have treatment." It was the first time in well over 12 years I had questions answered, I had things explained to me. I felt like I could breathe.

Steve:

When this started, we went to Cleveland every 3 weeks. It's a 3 ½-hour drive. And the worst part of that was when she probably should have been laying down somewhere sleeping, she was in the passenger seat of a car.

Brigitte:

I heard about WAINUA because I belonged to an amyloid support group. Went to my doctor’s and I told him how my other drug, I just didn't feel like it was working, I was having a lot of breakthrough symptoms, more neuropathy issues. And right away, before I even brought it up, he said, "WAINUA." He thought it was a very good option for me. I can give it to myself, I don't need a home health professional, I don't have to go somewhere.

Steve:

That's a big part of her life is to be able to once a month use this autoinjector instead of traveling.

Brigitte:

I like having control, I like taking charge, and for me, it's great. I do have my husband give it to me when he's home and available. So if he's around, he'll do it in the back of 1 of my arms, and if he's not, I'll do 1 leg, a leg, either side of my abdomen.

Steve:

Since it's a pressure-activated device, you just push it against the arm and wait the amount of time that it tells you, you hear the click when the needle withdraws and you're good to go.

Brigitte:

My doctor talked to me about possible side effects. My side effects were very mild lightheadedness, very mild nausea, and were gone the very next morning and I have not had any with my monthly treatments. And this may be different for others. It's just nice to be in control of my polyneuropathy symptoms. With WAINUA, I can feel it working and my symptoms are lessened. I don't have the continuous burning, the pins and needles, the numbness going through my hands, my lower extremities. I feel better between doses and I've told my girls, "God forbid if either one of you are positive. There is treatment to slow the process down. Don't let the disease take over you. You take control of the disease." What I would tell other patients diagnosed with this, get on treatments, get on WAINUA, and if you're having the similar symptoms, push for the testing. It's a simple blood test, any doctor can order it. Be your own advocate. Be strong. Push forward. With WAINUA, I can take my dog on a long walk, I'm no longer missing my family functions, I'm able to instruct my fitness classes. It's been wonderful.

Tom:

I'm Tom. I'm 58 years old. I work for my local county parks and rec department. And I am married and have 2 grown sons.

Ryan:

I'm Ryan.

Reese:

I'm Reese.

Ryan:

And we are Tom's sons. Our dad is like a handyman. He'll do everything for you. He helps me keep my car rolling.

Reese:

He's always happy to help us. You can tell that we all enjoy spending time together.

Tom:

In 2022, I was diagnosed with polyneuropathy caused by hereditary ATTR amyloidosis. When the hATTR-PN first manifested itself, I was walking to my kitchen to get my coffee, and I couldn't pick up my right foot. It was dragging. I'd be driving my car, my leg would cramp up. Sometimes, my, my fingers would lock up. I would get dizzy, I would lose my balance.

Reese:

Stairs were really tough for him. He would have to grab the handrail, walk down 2 feet at a time, breathe halfway down. It, it was rough.

Tom:

I was getting into a lot of gastrointestinal issues. There were some moments I didn't know if I was actually going to make it to the bathroom or not. And it just got to a point where I just didn't want to leave the house. I went through multiple doctors. I mentioned to them that my younger brother has this disease. He has hATTR-PN. He was diagnosed with it. His legs would not support his body weight. He eventually became bound to a wheelchair. So, I knew just from seeing him what possibly was ahead for me.

Ryan:

The hardest part of this whole thing was just getting, getting the diagnosis and actually figuring out what it was that was wrong with him.

Tom:

Fortunately, I did find a cardiologist that knows about hATTR. He said, "With the polyneuropathy amyloidosis, it affects your whole nervous system. That's why you have the GI issues, and that's why you get dizzy." So, I got the genetic test, came back positive. And, um, I was officially diagnosed in December 2022. With my boys, we had mentioned that it's hereditary, and I felt that they should get tested.

Ryan:

Both of us tested positive for it, and right now, luckily for us, it hasn't affected our day-to-day too much.

Reese:

And seeing the medical advancements that have just happened in the last 5, 6 years, it made it easy for me to get tested.

Tom:

I heard about WAINUA from my cardiologist. My doctor mentioned, um, a few side effects with it. The main one was possible vomiting. I haven't experienced any side effects, but that might not be what everyone else experiences. Since I've been on the WAINUA, I'm able to slowly get back to doing the things that we used to do.

Ryan:

It seems like it's kind of leveled him out versus kind of on a downward trajectory.

Reese:

The drop foot has not progressed anymore. His lower back pain seems to not come as often.

Tom:

The biggest thing that it has helped me with is the diarrhea, the gastrointestinal. That has been just a tremendous help just to be able to rest and not have to be up all night. It has enabled me to at least do the simple things at work, especially with, with the dexterity in my fingers. For me, the biggest decision to start using WAINUA was the fact that you could self-administer. You just pop it right on a fatty part of the thigh or the abdominal area, and you wait 10 seconds. It dispenses the medication, and that's it. You're done. Good to go. I inject it every fourth Tuesday. I supplement vitamin A now because of the treatment that I'm on. I am enrolled in WAINUA WAY. It was just unreal the support that I was receiving. My Patient Education Manager is phenomenal. She's always checking in with me, so I would definitely recommend WAINUA to anybody that has hATTR-PN.

Tom:

I obviously, I still have limitations, but I'm able to do the things with my boys that we did when they were younger.

Ryan:

Watch baseball, watch football, just go out in the yard and just mess around.

Reese:

Baseball was a huge part of our lives.

Tom:

We would go out and have a catch or go to the batting cages.

Reese:

Definitely brings huge peace of mind to us knowing that this medication is definitely helping.

Tom:

This is going to be a new path, a new journey, and being on WAINUA, it's able to help me navigate that much better.

Ryan:

Knowing that there's treatments like WAINUA out there is a complete game changer for me. If there weren't treatments, if there was nothing to do, I probably would not have gotten tested.

Tom:

Living with hATTR-PN has been a challenge for me, but WAINUA has definitely made it a lot more manageable. It has started to allow me to kind of get back to a normal life.