Hear from others managing GL with Myalept

Dwanna lives with GL, is taking Myalept, and has agreed to share her personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

Back and forth to the hospital.

When I was born, my mother noticed my skin wasn’t smooth like other babies and was leathery to the touch. I didn’t have any baby fat anywhere on my body, including my face. She knew right away that there was something different with me. She immediately had me checked by the doctor, but no matter what, my diagnosis was inconclusive. 

Growing up, I was much taller than my classmates, muscular, and I had very large hands and feet. It was because of my GL but we didn’t know it. Having this strange condition, my mother took me back and forth to the hospital for numerous tests. I dreaded those appointments. But apart from dealing with the bullies in grade school who made fun of my appearance calling me “skinny” and “muscle-bound,” I had a pretty normal life. I was very strong and athletic. I became very good at sports and enjoyed playing just about everything.

Finding a name for it.

While I was very active, there was something happening with my bones that I didn’t realize until I fell and broke my elbow. An MRI confirmed my bones were showing advanced bone age. I had to stop participating in sports, and shortly after was diagnosed with diabetes, which I now know was from the lack of leptin in my system. 

Without leptin, I was also always hungry and constantly eating, my blood sugar and triglycerides were often very high, and even though I was taking insulin, my diabetes was extremely insulin-resistant. Finally, when a doctor who was treating me realized I’d been seen at that same hospital years earlier, he checked my medical records and saw “lipodystrophy” written on my chart. He had me tested for it, and the results came back with a diagnosis of congenital generalized lipodystrophy. After 16 years, I finally had a name for it.

Feeling fortunate.

In 2000, my doctor approached my mother about having me participate in a clinical trial for a leptin replacement therapy, later called Myalept, and we all agreed. 

I’d been taking high doses of insulin for my diabetes because I was so insulin-resistant, but shortly after starting Myalept, my blood sugar was dropping so low I’d prop a chair in front of my refrigerator at night so I could eat immediately when I felt hypoglycemia coming on. Apparently, the leptin replacement was working to lower my blood glucose, so, because I was still taking insulin, I had to watch out for hypoglycemia. My doctor then told me I needed to reduce my insulin to avoid hypoglycemia. 

We quickly saw drops in my glucose and triglyceride numbers, and my doctors were very happy with my progress. My cholesterol levels came down, my triglycerides came down, and my diabetes was well controlled. The doctor said, “Wow! This medication works!” And I answered, “I’m glad something is working.” I also noticed a change in my appetite and I wasn’t as hungry as I was before. Of course, everyone is different, and their experiences with treatment may be different than mine.  

When I look back on my life, I feel fortunate that Myalept is available for people like me who are living with GL. 

Camerin’s daughter, Lorelei, lives with GL, is taking Myalept, and Camerin has agreed to share their personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

So many questions.

One day, when Lorelei was almost three years old, I noticed lumps on her forehead and her cheeks were rock hard. She was listless, tired, and running a fever. We ended up in the hospital for two weeks and every specialist we saw seemed to have no idea what Lorelei had. 

They did blood work and tests, and we found out she also had swollen lymph nodes all over her body and an enlarged liver and spleen. We later learned the fat cells in her body were hardening and she was diagnosed with panniculitis, which we were told was a symptom of something else. Within two months, all the fat in her body was gone and it’s never come back. 

We saw many different specialists each with their own possible diagnosis, but each test was negative and only left us with more questions.

A balancing act.

From the time Lorelei was three to five years old, she practically spent more time in the hospital than at home, being hospitalized a total of 13 times. It took almost a year, tons of blood work, and numerous biopsies to get a definitive diagnosis. But finally, she was diagnosed with acquired GL. 

Because of the lack of leptin caused by GL, she developed severe insulin resistance and diabetes, which made a complicated situation more difficult. And without leptin, her blood glucose and triglyceride levels were very high. It also wasn’t until a scary episode of pancreatitis that we discovered her body didn’t process fat correctly. That’s when we, including the doctors, learned just how complicated GL could be. 

I came to understand that GL is a disease that causes a loss of fat tissue all over her body and causes a lack of leptin, which can lead to certain health problems. But since Lorelei still didn’t have enough leptin at that point because we hadn’t started her on Myalept yet, we struggled trying to get her triglycerides and blood glucose levels down, and she was hospitalized many times.

Living her life.

We saw many more specialists – gastroenterologists, rheumatologists, endocrinologists, a pediatric hepatologist. We learned about treatment options and about Myalept, a leptin replacement therapy which was then in clinical trial. It was explained to us how, together with diet, Myalept could help treat complications from not having enough leptin by helping to lower her high blood glucose and high triglycerides. 

Before starting Myalept, we were trained by her nurse on how to give her injections. Lorelei had a skin reaction to her first Myalept injection, but she’s been able to take her injections well ever since. And the best part was that we saw results within months. Her triglycerides and blood glucose numbers got better. Of course, this has just been her experience. Also, before Myalept, Lorelei was a voracious eater. After she started Myalept, this all changed and we had to ask her if she was hungry because we were so used to her eating all the time.

Lorelei is an amazing little girl. I think sometimes adults pity themselves so much when they have a disease, yet she just runs around, plays, and lives her life. Of course, she still gets sick sometimes and has a lot going on with shots and medicines every day, but she’s a happy little girl, and I’m a proud momma!

Jason’s son, Troy, lives with GL, is taking Myalept, and Jason has agreed to share their personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

A growing problem.

Troy was around five years old when he started getting very unusual hives. Some days were just little ones on his arms, legs and stomach, but as they worsened, they started to show up on his face too. The hives were extremely itchy and once I had to rush him to the hospital since they nearly cut off his breathing. Troy also started losing fat in his cheeks and developed really muscular arms, even though he wasn’t working out. 

It wasn’t until a few years after that he started losing fat all over his body and we knew something was seriously wrong. He was also always voraciously hungry – but we didn’t understand back then it was because he didn’t have enough leptin in his body. 

By the time he was nine or 10, his belly started enlarging, and we learned his liver was enlarged and he had cirrhosis of the liver. Troy also developed diabetes. But at the time, we didn’t understand what was going on. 

One doctor after another.

Trying to get answers we’d go to the hospital and bounce around seeing multiple doctors – first the pediatrician, then an allergist, a pediatric liver specialist, foot specialist, rheumatoid arthritis specialist, an infectious disease doctor, gastroenterologist, and finally, an endocrinologist. 

It wasn’t until Troy was 10 that he was diagnosed with autoimmune hepatitis and acquired generalized lipodystrophy. But I didn’t believe the GL diagnosis at first – Troy had a lot of doctors tell him what he’d had before, and they’d all been wrong. Because of that, we refused treatment for two years, and his condition kept getting worse. 

Not giving up.

I talked with Troy’s doctors about getting into the clinical trial for Myalept, and while I had concerns, we knew he had to start treatment. We learned that some of Troy’s health problems were related to lack of leptin in his body because of his GL. So we realized Troy needed to add Myalept to his treatment plan to replace the leptin that he was missing. 

When Troy started Myalept, he was thinking it was one more shot he had to get that wasn’t going to work. At first he’d feel a burning sensation in his abdomen and wanted me to stop his treatment, but eventually, the pain lessened. His appetite got closer to normal, and not before long his triglyceride levels were lower, and his blood sugar level and A1C came down as well. 

Now Troy’s going into the 10th grade, and starting a job to save for a car. Through all this, we’ve learned life isn’t free, it’s a battle every day. But you don’t give up and keep your wishes and prayers alive. 

Dena lives with GL, is taking Myalept, and has agreed to share her personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

Thinner and thinner.

When I was baby and in pre-school, I was thin but still looked pretty normal. But starting in elementary school, I began to get thinner. I didn’t think much about it, but my mom worried. 

I think once I started going away to camp, I started getting asked questions like “Why are you so skinny?” At that point, I was only growing taller – I was the tallest in my class – but not really gaining weight. Since I also have celiac disease, for years we thought my thinness was related to the celiac disease. We thought that was the only concern.

Not just diabetes.

Looking back now, I had a lot of the signs of GL. I was so thin, and my face was becoming gaunt. You could easily see the veins on my arms and legs. I was hungry all the time too. I would regularly eat seconds and thirds of meals. 

I’d get occasional blood tests for my celiac disease, and on one of the visits we found my triglycerides were really high, and so were my liver enzymes and blood glucose. My pediatrician was concerned and referred me to a hepatologist. Then there was the geneticist, gastroenterologist, and endocrinologist too. I was diagnosed with Type 1 diabetes. But we later learned I was insulin resistant, which is not what typically happens with Type 1 diabetes. And in the sixth grade, the answer finally came. After many doctor’s appointments, we finally learned I didn’t just have diabetes, I had GL. 

Good results.

We found out that because I have GL, my body was missing leptin, which was why my triglycerides and blood sugar were so high – and that’s why I was insulin resistant too. When my mom understood what was at stake from a health standpoint, she knew getting on Myalept, which was then in clinical trial, to replace my missing leptin was something I had to do. She was relieved something could help me.

To me, maybe because I was pretty young, it seemed like just another shot I had to take. I remember when I was getting ready to go away to camp, I was nervous about doing it myself, but my mom, who was trained by the nurse, would guide me until eventually I could do it on my own. 

Once on Myalept, my complications from not having enough leptin began to improve – my triglycerides, blood sugar, and A1C all went down, and I started becoming less insulin resistant. My doctors were all pleased about that. I did lose a little weight after I started taking Myalept. We realized that Myalept wouldn’t help replace my missing fat, but it would help with complications due to the lack of leptin caused by having GL.

I’ve always been good about taking my shots when needed – even though sometimes it’s hard when I’m with friends and have to step away to do it. I also have to watch what I eat a little more. But I get regular checkups, and overall my numbers have been very good since I started on Myalept. 

Jilandre’s daughter, Raeya, lives with GL, is taking Myalept, and Jilandre has agreed to share their personal experience with Myalept and GL. Before taking Myalept, always ask your doctor about the benefits and risks of the treatment. Individual results may vary.

I knew my baby was different.

Raeya was born with congenital generalized liposdystrophy, even though we didn’t know it at the time. But as soon as she was born and handed to me, I knew there was something to be concerned about. 

Her skin was tight – not like other newborn babies I’d seen. She was also very muscularly defined, even though her belly was round. She looked like a bodybuilder. 

Things got worse before they got better. 

Shortly after her birth, Reaya became jaundiced, which was confusing since her appetite seemed almost insatiable. She was always hungry. Then, when she was about two months old, Raeya’s blood work revealed high triglycerides, high cholesterol, and high blood pressure. Basically, this tiny baby with no fat had the blood work of an obese adult. 

We then saw a gastroenterologist and found out that her liver was enlarged, which explained the size of her belly. She also had advanced bone age. We went back and forth seeing probably eight different specialists. It wasn’t until a geneticist we’d seen went to a seminar about lipodystrophy that suddenly all of the questions and concerns about Raeya made sense. Finally, when she was five months old, she was diagnosed with congenital generalized lipodystrophy. 

We were finally able to give her disease a name and get some answers. We learned that Raeya’s body doesn’t produce enough leptin, which tells the brain how much fat to store and where to store it, among other things. And the lack of leptin in Raeya’s body was causing her high triglycerides and high blood sugar, as well as other complications.

Seeing results.

We talked to Raeya’s doctors about treatment options, and found out there was a clinical trial for a leptin replacement therapy for generalized lipodystrophy. With her doctors, we started Raeya on the medication, now called Myalept. Her appetite was the first thing to change. When she ate, she wouldn’t eat everything, and eventually, she ate less often too. The change was overwhelming because Raeya went from an insatiable appetite to me constantly asking her if she was hungry and forcing her to eat.

I knew diabetes could be a complication of GL, but fortunately, we never saw this with Raeya. Since starting Myalept, her blood glucose has remained within the normal range. And her triglycerides came way down.

Giving Raeya the Myalept injection was difficult at first because of her lack of fat, but over time it got easier. The nurses trained us in how to mix the medication and give the shot, then it was our turn. It was a two-person job the first time. Raeya says she used to be afraid, but now it only hurts sometimes. In fact, now she’ll walk up to me when it’s time for her shot. I’ve encouraged her to get involved and I give her some choice about where she gets the injection – she’ll put her finger right over the spot where I should do it so she feels more in control.   

Raeya’s day-to-day life is a lot like any other kid, but she gets a shot every morning. She’s a bubbly, full-of-energy kid. Every day she gets up and lives her life like nothing is holding her back.