Hear from others with GL

Learn from the experiences of real people with GL –
and get insights from parents of children with GL.


Select Important Safety Information

Myalept is only available through a restricted program called the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Your doctor must be enrolled and certified in the program in order to prescribe Myalept.

You should not take Myalept if you have general obesity not caused bt a congenital leptin deficiency; are allegic to metreleptin or any of the ingredients in Myalept. Symptoms of an allergic reaction include rash, itching (hives), swelling of face, lips, tongue, or throat, problems breathing or swallowing, fainting or dizziness, rapid heart beat.

Dwanna

Dwanna

  • Lives in Texas
  • Has congenital GL
  • Taking Myalept since 2000
After I started taking Myalept, my triglyceride levels started going down.

Read Dwanna's story

Select Important Safety Information
Before using Myalept, tell your doctor if you have any medical conditions including if you are pregnant or plan to become pregnant as it is unknown if Myalept will harm your unborn body. If you become pregnant while using Myalept, talk to your healthcare provider about registering with a program to collect information about the outcomes of moms and babies exposed to Myalept during pregnancy. You can enroll in the Myalept program by calling 1-855-669-2537 are planning or plan to nurse. You should not nurse while you take 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. When I broke my elbow, I had to stop participating in sports.

Finding a name for it.

Later, I was diagnosed with diabetes, which I now know was from the lack of leptin in my system. 

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. 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. 

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Camerin and Lorelei

Camerin and Lorelei

  • Live in California
  • Camerin’s daughter, Lorelei, lives with acquired GL
  • Taking Myalept since 2012
Once Lorelei started Myalept to replace her missing leptin, her triglycerides and blood glucose numbers got better.

Read Camerin and Lorelei's story

Select Important Safety Information
Before using Myalept, tell your doctor if you have any medical conditions including if you have or have had problems with your blood cells, including low blood cell counts (especially your white blood cells), bone marrow, immune system, pancreas, swollen lymph nodes, lymphoma, high triglyceride levels, or use insulin or sulfonylurea.

 

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 fat and 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. Lorelei didn’t have enough leptin and 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 may help 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.

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!

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Jason and Troy

Jason and Troy

  • Live in Michigan
  • Jason’s son, Troy, lives with acquired GL
  • Taking Myalept since 2010
When Troy started losing fat all over his body, we knew something was seriously wrong.

Read Jason and Troy's story

Select Important Safety Information
Myalept may worsen symptoms caused by certain problems in your immune system (autoimmune disorder). Ask your doctor about what symptoms you should watch for that may require further testing. Talk to your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of Myalept. For more information, speak to your doctor or pharmacist. Tell your doctor about all the medications you take, including prescription and nonprescription medicines, vitamins, and herbal supplements. Take Myalept exactly as your doctor instructs you.

 

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.

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. 

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. Not before long his triglyceride levels were lower, and his blood sugar level and A1C came down as well. 

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. 

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Dena

Dena

  • Lives in New Jersey
  • Has congenital GL
  • Taking Myalept since 2009
Looking back now, I had a lot of the signs of GL

Read Dena's story

Select Important Safety Information
The most common side effects of Myalept include headache, low blood sugar, decreased weight, and/or abdominal pain.

 

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. 

My results.

We found out that because I have GL, my body was missing leptin, which was contributing to 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. 

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 these 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. 

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Jilandre and Raeya

Jilandre and Raeya

  • Live in Maryland
  • Jilandre’s daughter, Raeya, lives with congenital GL
  • Taking Myalept since 2010
Leptin is a hormone that Raeya’s body doesn’t make enough of, and Myalept is a leptin replacement therapy, so it made sense for Raeya to start Myalept right away.

Read Jilandre and Raeya's story

Select Important Safety Information
Myalept is only available through a restricted program called the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Your doctor must be enrolled and certified in the program in order to prescribe 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. 

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 and high blood sugar. 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. And the lack of leptin in Raeya’s body was contributing to her high triglycerides and high blood sugar.

What happened next.

We talked to Raeya’s doctors about treatment options for patients with GL, and found out there was a clinical trial for a leptin replacement therapy. With her doctors, we started Raeya on the medication, now called Myalept.

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.

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We’re here to help

Whether you want more information, have questions, or are looking for resources and support, we're by your side. Connect with ByMySide to learn more.

To speak with a ByMySide Coordinator, call 1-855-6MYALEPT (1-855-669-2537), Monday through Friday, 8 am to 8 pm ET.

ByMySide is a personalized support program for Myalept patients.  When you complete the Patient Authorization Form for the ByMySide program you can access additional support services and resources at no cost to you.  These are in addition to the support you are currently receiving in regard to your Myalept therapy through your dedicated nurse case manager including insurance investigation financial assistance (for eligible patients), 24/7 nurse support, nurse education and injection training, dietician support, and refill reminders. 

Risk of Neutralizing antibodies and risk of lymphoma.

Important Safety Information

Myalept is only available through a restricted program called the Myalept Risk Evaluation and Mitigation Strategy (REMS) Program. Your doctor must be enrolled and certified in the program in order to prescribe Myalept.

Myalept may cause serious side effects, including:

  • risk for developing certain proteins called neutralizing antibodies that may reduce how well your own leptin or Myalept works. Side effects of these antibodies may include infection, problems with blood sugar (including diabetes), or an increase in triglycerides
  • increased risk of a type of blood cancer called lymphoma

You should not take Myalept if you:

  • have general obesity not caused by a congenital leptin deficiency
  • are allergic to metreleptin or any of the ingredients in Myalept.  Symptoms of an allergic reaction include rash, itching (hives), swelling of face, lips, tongue, or throat, problems breathing or swallowing, fainting or dizziness, rapid heartbeat.

Before using Myalept, tell you doctor if you have any medical conditions including if you:

  • have or have had problems with your blood cells, including low blood cell counts (especially your white blood cells), bone marrow, immune system, pancreas, swollen lymph nodes, lymphoma, high blood triglyceride levels, or use insulin or a sulfonylurea
  • are pregnant or plan to become pregnant as it is unknown if Myalept will harm your unborn baby. If you become pregnant while using Myalept, talk to your healthcare provider about registering with a program to collect information about the outcomes of moms and babies exposed to Myalept during pregnancy. You can enroll in the Myalept program by calling 1-855-669-2537
  • are nursing or plan to nurse. You should not nurse while you take Myalept

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Other possible side effects

The most common side effects of Myalept include headache, low blood sugar, decreased weight, and/or abdominal pain.

For newborns and infants, mix Myalept with sterile water for injection (preservative-free) (WFI). Serious side effects including death have happened in newborns or infants who have received the preservative benzyl alcohol.  Bacteriostatic water for injection contains benzyl alcohol and it should not be used to mix Myalept for newborns and infants.

You may get low blood sugar (hypoglycemia) if you take Myalept with other medicines used to lower blood sugar, such as insulin or sulfonylurea. Your doses of these medications may need to be lowered while you use Myalept. Tell your doctor right away if you experience shakiness, sweating, headache, drowsiness, weakness, dizziness, confusion, irritability, hunger, fast heartbeat, or a jittery feeling because these may be signs of low blood sugar (hypoglycemia).

Myalept may worsen symptoms caused by certain problems in your immune system (autoimmune disorder). Ask your doctor about what symptoms you should watch for that may require further testing.

Talk to your healthcare provider about any side effect that bothers you or that does not go away. These are not all the possible side effects of Myalept. For more information, speak to your doctor or pharmacist. Tell your doctor about all the medications you take, including prescription and nonprescription medicines, vitamins, and herbal supplements.  Take Myalept exactly as your doctor instructs you.

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.

This is the most important information about Myalept. For more detailed information, Please see the Medication Guide, Instructions for Use and full Prescribing Information including Box Warning.

What is Myalept?

Myalept (metreleptin), a prescription medication, is a leptin replacement therapy used with a doctor-recommended diet to treat problems caused by not having enough leptin (leptin deficiency) in people with generalized lipodystrophy.

  • It is not known if Myalept is safe and effective when used to treat problems (complications) caused by partial lipodystrophy or to treat liver disease, including non-alcoholic steatohepatitis (NASH)
  • Myalept should not be used to treat people with HIV-related lipodystrophy or people with metabolic disease, including diabetes mellitus and hypertriglyceridemia, without signs or symptoms of congenital or acquired generalized lipodystrophy.

Please see the Medication Guide, Instructions for Use and full Prescribing Information including Box Warning.

 

Photographs on this website feature people with GL and their caregivers.