ELELYSO Patient Stories
ELELYSO® (taliglucerase alfa) for injection patients have shared their stories about living with and managing Gaucher disease with ELELYSO. Listening to their experiences may help you or a loved one stay informed.
We asked patients to send us their stories about ELELYSO. These are their stories.
Dave
Lawndale, CA
“The one thing that I really hope you take away from my experience with Type 1 Gaucher disease is to not let it define your personality and life.”
Key Facts:
- Age: 59
- Environmental consultant and independent business owner
- Diagnosed with Type 1 Gaucher disease in 2004
Dave had a healthy childhood by all accounts. When he was in his mid-40s with his platelet counts consistently reading too low and his bilirubin reading too high, he went to see a hematologist. Ultimately a bone marrow biopsy provided a diagnosis—Type 1 Gaucher disease.
It took Dave some time to come to terms with his diagnosis. He started educating himself and talking to his doctor about what treatments were available. At that time, Dave began enzyme replacement therapy. Later, Dave heard about ELELYSO and spoke to his doctor about his treatment plan. Dave began treatment with ELELYSO and has been managing his condition. He also focuses on his new passion: raising awareness about Gaucher disease.
Dave now works hard to help others with Type 1 Gaucher disease understand the importance of advocating for themselves and not letting Gaucher define their lives.
Donald
Alexandria, AL
“Type 1 Gaucher disease is so rare that the only time some of us have an opportunity to meet someone else with Gaucher is at an infusion center or a Gaucher disease event. I hope patients continue to take advantage of these opportunities.”
Key Facts:
- Age: 75
- Retired business owner
- Diagnosed with Type 1 Gaucher disease in 1978
Don was born and raised in Alabama, and over the years he has worked retail and industrial jobs. Now that he’s retired, he has more time to spend with his daughter and 2 granddaughters, one of whom lives with him. Don also loves to play duplicate bridge and is the club manager for his local bridge club.
He felt that he could contribute to the advancement of Type 1 Gaucher disease clinical research by volunteering for research studies. When he learned of a clinical trial for a treatment, now known as ELELYSO, he eagerly volunteered. He is now treated with ELELYSO and continues to keep his health care team in the loop about how his treatment is going.
Don encourages other people living with Type 1 Gaucher disease to find a doctor who understands it, has other Gaucher patients, and is familiar with all of the treatment options. He is thankful for the opportunity to have taken part in clinical research and to be on a treatment that helps him manage his Type 1 Gaucher disease.
Martin
Dallas, PA
“All of us encounter obstacles in our lives that we never expected—some we can control, some we can’t. If I’ve learned anything, it’s that I can’t afford to dwell on the negatives or mistakes I’ve made in the past.”
Key Facts:
- Age: 70
- Retired physician
- Diagnosed with Type 1 Gaucher disease in 1971
Martin lived a relatively normal life until he went away to college. As a freshman, he started experiencing symptoms of Type 1 Gaucher disease. The school’s infirmary immediately sent him to be admitted and found that he had anemia and an enlarged spleen.
One of Martin’s colleagues at a medical center took it upon himself to figure out what was wrong with him, and Martin was ultimately diagnosed with Type 1 Gaucher disease. Coincidentally, his roommate at the time was studying this rare lysosomal storage disorder.
Treatment wasn’t available at the time, so Martin continued on with his life and struggled as his symptoms got worse. He started taking painkillers, which led him down a self-destructive road. Eventually, Martin became addicted. When a treatment became available, he jumped at the chance to start it. Years later, he was able to overcome his addiction to painkillers with the help of a 12-step program.
A supply shortage of his treatment caused Martin to look for a new option. His physician recommended joining a clinical trial for a treatment now known as ELELYSO. One thing that appealed to Martin, because of his Jewish heritage, was that the manufacturer was based in Israel. ELELYSO helped him manage his symptoms, so he continued with it after the trial concluded. He has been using the Gaucher Personal Support program that Pfizer offers as part of their commitment to the Gaucher community. Martin continues to take ELELYSO every 2 weeks, knowing how important it is that he stay on treatment.
Michele
Selinsgrove, PA
“We are privileged to have so many people who are devoted to improving the lives of people in the Gaucher community.”
Key Facts:
- Age: 40
- Homemaker, previously an occupational therapist
- Diagnosed with Type 1 Gaucher disease in 1985
Michele is a mother of 2 pre-teens and 1 teenager. If that doesn’t keep her busy enough, she enjoys several creative pursuits, like painting, writing, and music. In fact, she directs a children’s theater program each summer.
When Michele was young, she was often sick, and her mom was concerned about her distended abdomen and suspected that she was not well. Some doctors dismissed Michele’s symptoms; others suggested that it was leukemia, but no one had concrete answers. Finally, Michele was connected to a new hospital and was diagnosed with Type 1 Gaucher disease when she was just 7 years old.
One of Michele’s biggest challenges has been managing some of her Type 1 Gaucher disease symptoms without pushing herself too hard. Thankfully, with ELELYSO, Michele has been able to manage her condition.
Michele has learned the importance of asking for help, advocating for herself, and utilizing resources, like Pfizer’s Gaucher Personal Support offerings.
Michelle
Albany, OR
“There isn’t a cure for Type 1 Gaucher disease, so I am very thankful that there are treatments.”
Key Facts:
- Age: 45
- Diagnosed with Type 1 Gaucher disease in 2015
- Provides ethics oversight for a health company
As a child, Michelle found it difficult to engage in the same activities as other children. That made it hard for her to play like other children would. Doctors were unable to find exactly what was ailing her.
As an adult, Michelle is a busy wife and mom of 2 active boys. Discomfort in her abdomen eventually led to the removal of her spleen and gallbladder.
It was a specialist trying to help Michelle who ordered a bone marrow biopsy that confirmed Type 1 Gaucher disease. For Michelle, her diagnosis offered a sense of relief and confirmed that she hadn’t been “crazy” all those years.
Michelle and her specialist discussed a number of different treatment options, and together they decided to initiate treatment with ELELYSO.
Now Michelle is an active member on many Gaucher-related social media sites. She’s focused on raising awareness of Gaucher disease and helps provide support and resources to newly diagnosed individuals.