video_id
6358151992112
account_id
1852113022001
title
Tim’s story
poster
See Tim’s full story
transcript
00:11
Tim
This is the last time that I had to infuse myself with factor before I got BEQVEZ. Ooh. This is actually me getting BEQVEZ. You can see even behind the mask. I am very excited.
00:23
Tim
Okay.
00:24
Tim
Hey, I’m Tim. I got BEQVEZ back in 2021. Let me tell you all about it. Oh, there it is. There’s me.
00:35
Tim
BEQVEZ is a one-time gene therapy for certain adults with moderate to severe hemophilia B. Your doctor will perform certain tests and review your medical condition and history to determine if BEQVEZ is right for you.
00:47
Tim
A lot of my childhood was just in and out of the hospitals. Eventually, I started on a prophylactic schedule. But then there’s all the logistical planning that comes along with having hemophilia. So much of my time was dedicated to getting the treatment, ordering the factor, making sure that I had it on hand, and it's stored properly, kept at the right temperature. It was just the biggest headache.
01:07
Brit
Hey Bub, wanna dye your hair?
01:09
Tim
Yeah.
01:09
Brit
Alright, let’s go. You gotta take this off... you're gonna get dye on it.
01:12
Tim
Yes, yes, yes.
01:12
Tim
Yes. Having hemophilia, it’s more than just about you. It’s about your family...
01:18
Brit
Woooo. Alright! Got a towel here for you.
01:21
Tim
…which I have right here.
01:21
Tim
My beautiful wife, Brittany.
01:23
Brit
Hey. A little before picture…
01:30
Tim
Alright, let’s do it.
01:31
Brit
Let’s do it.
01:32
Tim
Through my twenties and even early thirties, I just was constantly neglecting my hemophilia against my doctor’s advice. When my band became a little bit more serious, it involved a lot of travel. In my head, I'm band first, music first. If I can’t treat today, I’ll treat tomorrow. We were traveling in a van all across the country. There was many times where I was in the back of the van infusing myself right before a gig.
02:02
Brit
Let me see ya.
02:03
Tim
I was missing a lot of treatments because I was just too busy. And then I would always pay the price. And I would have breakthrough bleeds in my hip caused by my mismanagement of my hemophilia.
02:13
Brit
Here. Come over here. What’s dad doing?
02:20
Tim
There’s always been that little thing in the back of my head. One day, maybe science will be far enough advanced that I can just do this one treatment, and I can just live my life. As my doctor informed me about the possible results from the gene therapy, he said, this is not a cure. It’s not a cure for hemophilia. It’s gonna work differently for everyone. And it’s a one-time thing.
02:47
Tim
He also told me he’d let me know when I could stop factor IX prophylaxis or resume it, and whether I’d need to take FIX for surgeries, injuries, or bleeds. My doctor advised about NAb testing and liver health tests. We also talked about the importance of not donating sperm and being abstinent, or using a condom for the first six months after the infusion, and limiting alcohol during the first year.
03:12
Tim
I said, let’s do it. I’m ready to change my lifestyle. I was just…just kind of daydreaming about my future. So if you have any hesitation with getting BEQVEZ, I would recommend talking to…
03:35
Tim
…your doctor to help determine if BEQVEZ is right for you.
03:40
Tim
The care team was super supportive and they planned the entire thing out so well for me, and they were so…
03:46
Tim
…hospitable to our schedules. They just gave me a timeline of when my blood draws were gonna be. Once or twice a week at first, and over time, less and less.
03:57
Tim
My doctors were super prepared and knew of the possible side effects. I personally didn't have any side effects, but some people who took BEQVEZ needed corticosteroids to help manage elevated liver enzymes. My doctors always told me I still have to have my factor on hand, I still have hemophilia. Just for me personally, it was a lot more mild hemophilia.
04:16
Tim
It’s not possible to predict who will respond to BEQVEZ and how long it will last.
04:22
Tim
Since BEQVEZ, I’ve had zero bleeds and zero regrets. My factor levels have been mild. Oh, that’s when we were exploring the temple in Tokyo. So I had my first a-ha moment about a year after getting BEQVEZ. I was on tour with my band in the UK, and of course something happened where I, like, stubbed my toe or hurt myself. So immediately my brain goes, great. Now I have to deal with factor and treating myself so I’m not, you know, bleeding in a foreign country. But then I had this moment where I’m like, I don’t have to treat myself.
04:55
Tim
I didn’t realize how much of a time commitment it really was until I just didn’t have to deal with all this stuff anymore.
05:06
Tim
It’s like I took hemophilia from ten and just turned it down on the volume notch. What was that, Nacho? Oh, Nacho says, talk to your doctor and see if BEQVEZ is right for you. Alright, from the top. Tippity top! This one’s for all the people out there living with hemophilia. To just take all that, just everything related to hemophilia and just make it just this thing that’s in the back of your mind.
05:41
Tim
For me, that means the world.
05:48
Tim
This is me living with hemophilia differently.
05:52
Tim
Keep watching for Important Safety Information and see the full Prescribing Information on BEQVEZ.com.
05:57
Voiceover
Indication and Important Safety Information for BEQVEZ (fidanacogene elaparvovec-dzkt).
What is BEQVEZ? BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, or have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds. Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for neutralizing antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ. Important Safety Information. Before receiving BEQVEZ, tell your healthcare professional about all your medical conditions, including if you have kidney or liver problems, including hepatitis, have factor IX inhibitors or a history of factor IX inhibitors, have an active infection. BEQVEZ may cause serious side effects, including increased liver enzymes.
06:51
Voiceover
Most patients treated with BEQVEZ developed elevated liver enzyme levels and most did not experience any symptoms. Your healthcare professional will monitor liver enzymes and factor IX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes. Limit alcohol consumption during the first year following BEQVEZ infusion, as alcohol may reduce the effect of BEQVEZ and may increase liver enzyme levels. Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include, but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache. BEQVEZ can insert itself into the DNA of cells in the human body.
07:45
Voiceover
The effect that insertion may have on those cells is unknown but may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with BEQVEZ. The most common side effect of BEQVEZ is increased liver enzymes. These are not all the possible side effects of BEQVEZ. For more information, ask your healthcare professional. Talk to your healthcare professional before receiving any vaccinations if you are taking a corticosteroid. Talk to your doctor about any medications you plan to take including over-the-counter medications, herbal supplements, and vitamins as certain substances can affect the liver and may reduce the effectiveness of BEQVEZ. Your healthcare professional will test your factor IX activity levels and for factor IX inhibitors. After receiving BEQVEZ, your doctor will discuss whether and when you are able to stop prophylaxis,
08:28
Voiceover
if you need to resume prophylaxis, and actions you may need to take for surgeries, procedures, injuries, and bleeding events. Do not donate blood, organs, tissues, or cells for transplantation following administration of BEQVEZ. BEQVEZ is not intended for administration to women. Males should not donate sperm and should use a male condom or not have sexual intercourse for up to six months after receiving BEQVEZ. Patients and caregivers should ensure proper handling of any materials that have come into contact with a patient’s urine, feces, saliva, mucus, or semen in the first six months after BEQVEZ infusion. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800 FDA 1088. Please see full Prescribing Information at www.BEQVEZ.com.
09:17
Tim
Talk to your doctor about BEQVEZ.
Tim
This is the last time that I had to infuse myself with factor before I got BEQVEZ. Ooh. This is actually me getting BEQVEZ. You can see even behind the mask. I am very excited.
00:23
Tim
Okay.
00:24
Tim
Hey, I’m Tim. I got BEQVEZ back in 2021. Let me tell you all about it. Oh, there it is. There’s me.
00:35
Tim
BEQVEZ is a one-time gene therapy for certain adults with moderate to severe hemophilia B. Your doctor will perform certain tests and review your medical condition and history to determine if BEQVEZ is right for you.
00:47
Tim
A lot of my childhood was just in and out of the hospitals. Eventually, I started on a prophylactic schedule. But then there’s all the logistical planning that comes along with having hemophilia. So much of my time was dedicated to getting the treatment, ordering the factor, making sure that I had it on hand, and it's stored properly, kept at the right temperature. It was just the biggest headache.
01:07
Brit
Hey Bub, wanna dye your hair?
01:09
Tim
Yeah.
01:09
Brit
Alright, let’s go. You gotta take this off... you're gonna get dye on it.
01:12
Tim
Yes, yes, yes.
01:12
Tim
Yes. Having hemophilia, it’s more than just about you. It’s about your family...
01:18
Brit
Woooo. Alright! Got a towel here for you.
01:21
Tim
…which I have right here.
01:21
Tim
My beautiful wife, Brittany.
01:23
Brit
Hey. A little before picture…
01:30
Tim
Alright, let’s do it.
01:31
Brit
Let’s do it.
01:32
Tim
Through my twenties and even early thirties, I just was constantly neglecting my hemophilia against my doctor’s advice. When my band became a little bit more serious, it involved a lot of travel. In my head, I'm band first, music first. If I can’t treat today, I’ll treat tomorrow. We were traveling in a van all across the country. There was many times where I was in the back of the van infusing myself right before a gig.
02:02
Brit
Let me see ya.
02:03
Tim
I was missing a lot of treatments because I was just too busy. And then I would always pay the price. And I would have breakthrough bleeds in my hip caused by my mismanagement of my hemophilia.
02:13
Brit
Here. Come over here. What’s dad doing?
02:20
Tim
There’s always been that little thing in the back of my head. One day, maybe science will be far enough advanced that I can just do this one treatment, and I can just live my life. As my doctor informed me about the possible results from the gene therapy, he said, this is not a cure. It’s not a cure for hemophilia. It’s gonna work differently for everyone. And it’s a one-time thing.
02:47
Tim
He also told me he’d let me know when I could stop factor IX prophylaxis or resume it, and whether I’d need to take FIX for surgeries, injuries, or bleeds. My doctor advised about NAb testing and liver health tests. We also talked about the importance of not donating sperm and being abstinent, or using a condom for the first six months after the infusion, and limiting alcohol during the first year.
03:12
Tim
I said, let’s do it. I’m ready to change my lifestyle. I was just…just kind of daydreaming about my future. So if you have any hesitation with getting BEQVEZ, I would recommend talking to…
03:35
Tim
…your doctor to help determine if BEQVEZ is right for you.
03:40
Tim
The care team was super supportive and they planned the entire thing out so well for me, and they were so…
03:46
Tim
…hospitable to our schedules. They just gave me a timeline of when my blood draws were gonna be. Once or twice a week at first, and over time, less and less.
03:57
Tim
My doctors were super prepared and knew of the possible side effects. I personally didn't have any side effects, but some people who took BEQVEZ needed corticosteroids to help manage elevated liver enzymes. My doctors always told me I still have to have my factor on hand, I still have hemophilia. Just for me personally, it was a lot more mild hemophilia.
04:16
Tim
It’s not possible to predict who will respond to BEQVEZ and how long it will last.
04:22
Tim
Since BEQVEZ, I’ve had zero bleeds and zero regrets. My factor levels have been mild. Oh, that’s when we were exploring the temple in Tokyo. So I had my first a-ha moment about a year after getting BEQVEZ. I was on tour with my band in the UK, and of course something happened where I, like, stubbed my toe or hurt myself. So immediately my brain goes, great. Now I have to deal with factor and treating myself so I’m not, you know, bleeding in a foreign country. But then I had this moment where I’m like, I don’t have to treat myself.
04:55
Tim
I didn’t realize how much of a time commitment it really was until I just didn’t have to deal with all this stuff anymore.
05:06
Tim
It’s like I took hemophilia from ten and just turned it down on the volume notch. What was that, Nacho? Oh, Nacho says, talk to your doctor and see if BEQVEZ is right for you. Alright, from the top. Tippity top! This one’s for all the people out there living with hemophilia. To just take all that, just everything related to hemophilia and just make it just this thing that’s in the back of your mind.
05:41
Tim
For me, that means the world.
05:48
Tim
This is me living with hemophilia differently.
05:52
Tim
Keep watching for Important Safety Information and see the full Prescribing Information on BEQVEZ.com.
05:57
Voiceover
Indication and Important Safety Information for BEQVEZ (fidanacogene elaparvovec-dzkt).
What is BEQVEZ? BEQVEZ is a one-time gene therapy used for the treatment of adults with moderate to severe hemophilia B who are receiving routine prophylaxis, or have a current life-threatening bleed or a history of life-threatening bleeds, or have repeated serious spontaneous bleeds. Before treatment with BEQVEZ, your healthcare professional will conduct a blood test to check for neutralizing antibodies to the AAVRh74var virus. The results of this testing will help determine if you may receive BEQVEZ. Important Safety Information. Before receiving BEQVEZ, tell your healthcare professional about all your medical conditions, including if you have kidney or liver problems, including hepatitis, have factor IX inhibitors or a history of factor IX inhibitors, have an active infection. BEQVEZ may cause serious side effects, including increased liver enzymes.
06:51
Voiceover
Most patients treated with BEQVEZ developed elevated liver enzyme levels and most did not experience any symptoms. Your healthcare professional will monitor liver enzymes and factor IX activity levels before administration of BEQVEZ and frequently following the administration to detect and identify possible elevations in liver enzymes and to monitor your response to BEQVEZ. Your doctor may prescribe a corticosteroid for the treatment of elevated liver enzymes. Limit alcohol consumption during the first year following BEQVEZ infusion, as alcohol may reduce the effect of BEQVEZ and may increase liver enzyme levels. Infusion reactions, including hypersensitivity and severe allergic reactions (anaphylaxis) may occur. Alert your healthcare professional right away if you get any symptoms of hypersensitivity, which may include, but are not limited to low blood pressure, fever, heart palpitation, nausea, vomiting, chills, or headache. BEQVEZ can insert itself into the DNA of cells in the human body.
07:45
Voiceover
The effect that insertion may have on those cells is unknown but may contribute to a theoretical risk of cancer. There have been no reported cases of cancer caused by treatment with BEQVEZ. The most common side effect of BEQVEZ is increased liver enzymes. These are not all the possible side effects of BEQVEZ. For more information, ask your healthcare professional. Talk to your healthcare professional before receiving any vaccinations if you are taking a corticosteroid. Talk to your doctor about any medications you plan to take including over-the-counter medications, herbal supplements, and vitamins as certain substances can affect the liver and may reduce the effectiveness of BEQVEZ. Your healthcare professional will test your factor IX activity levels and for factor IX inhibitors. After receiving BEQVEZ, your doctor will discuss whether and when you are able to stop prophylaxis,
08:28
Voiceover
if you need to resume prophylaxis, and actions you may need to take for surgeries, procedures, injuries, and bleeding events. Do not donate blood, organs, tissues, or cells for transplantation following administration of BEQVEZ. BEQVEZ is not intended for administration to women. Males should not donate sperm and should use a male condom or not have sexual intercourse for up to six months after receiving BEQVEZ. Patients and caregivers should ensure proper handling of any materials that have come into contact with a patient’s urine, feces, saliva, mucus, or semen in the first six months after BEQVEZ infusion. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800 FDA 1088. Please see full Prescribing Information at www.BEQVEZ.com.
09:17
Tim
Talk to your doctor about BEQVEZ.