LIVING WITH TYPE 1 GAUCHER DISEASE: REAL STORIES, REAL CONVERSATIONS.

Discover what life with type 1 Gaucher disease (GD1) really looks like through the voices of those who live it every day.

Through a blend of roundtable discussions and personal interviews, you’ll hear authentic stories from our patient ambassadors, Roxanne and Wayne. They share their real-life experiences with GD1, including family planning and treatment with VPRIV.

Moderating the roundtable discussion is Dr. Eliana Fine, an obstetrician and gynecologist, offering expert insights on topics that matter most — such as genetic testing, disease monitoring, and women’s health.

This conversation offers both practical knowledge and heartfelt perspectives to help individuals and families navigate life with GD1 with confidence.

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Please keep watching for Important Safety Information.

Discover what life with type 1 Gaucher disease (GD1) really looks like through the voices of those who live it every day.

Through a blend of roundtable discussions and personal interviews, you’ll hear authentic stories from our patient ambassadors, Roxanne and Wayne. They share their real-life experiences with GD1, including family planning and treatment with VPRIV.

Moderating the roundtable discussion is Dr. Eliana Fine, an obstetrician and gynecologist, offering expert insights on topics that matter most — such as genetic testing, disease monitoring, and women’s health.

This conversation offers both practical knowledge and heartfelt perspectives to help individuals and families navigate life with GD1 with confidence.

Disease Education

15 Mins

chapter-1

GD1 Inheritance

00:00

3 minutes

chapter-2

Family Planning

00:00

3 minutes

chapter-3

Experiences With GD1

00:00

3 minutes

chapter-4

GD1 and advocating for care

00:00

3 minutes

chapter-5

Patient Diagnosis Stories

00:00

3 minutes

VPRIV Education

10 Mins

chapter-5

VPRIV Education

00:00

10 minutes

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Meet our ambassadors

Roxanne

Age at Diagnosis: 42

Location: Texas, US

Roxanne is a dedicated caregiver, community servant, and patient advocate living with type 1 Gaucher disease. Diagnosed in her early 40s, she navigated treatment and emotional challenges while continuing to support her family and others in the GD1 community.

Wayne

Age at Diagnosis: 15

Location: Massachusetts, US

Wayne is a long‑time advocate living with type 1 Gaucher disease since age 15. Now in his 70s, he balances his condition with an active personal, professional, and community life, sharing his journey to encourage others to be informed and proactive in their care.

Click on an ambassador to learn more about them

Meet Our healthcare professional

dr-eliana-fine

Dr. Eliana Fine, MD

Dr. Fine is an obstetrician-gynecologist dedicated to providing compassionate, comprehensive women’s healthcare. She completed residency training at Stony Brook University Hospital and Jersey Shore University Medical Center, and earned her medical degree from the Renaissance School of Medicine at Stony Brook University through an accelerated three-year MD program. Dr. Fine is also co-founder of the Jewish Orthodox Women’s Medical Association (JOWMA) and is deeply committed to advancing women’s health education and advocacy. As a physician and a mother of two, she brings both clinical expertise and personal perspective to her work, with a strong focus on patient-centered, individualized care.

For more resources on VPRIV and type 1 Gaucher Disease, please visit the links here:

VPRIV Clinical Study Information

VPRIV Clinical Study Information

View the clinical study data behind VPRIV.

View Page

VPRIV Discussion Guide

VPRIV Discussion Guide

Questions and discussion points to raise with your doctor, and a log to record your symptoms and treatments.

Download Guide

VPRIV Patient Brochure

VPRIV Patient Brochure

Learn all about VPRIV from treatment management to dosing information.

Download

Explore support organizations

Explore support organizations

Explore patient advocacy groups and resources offering education and support

View Page

important safety information

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat.

important safety information

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat. VPRIV should be administered under the supervision of a healthcare professional. Appropriate medical support should be available when VPRIV is administered.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the reactions were mild and, in patients not previously treated, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. After the drug was approved, additional hypersensitivity reactions of chest discomfort, difficulty breathing, itching and vomiting have been reported. In some cases, vomiting can be serious and require hospitalization and/or stopping the medication.

If anaphylactic or other acute reactions occur, seek immediate medical care. Your healthcare provider will immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment. A hypersensitivity reaction should be treated based on the severity of the reaction. Your healthcare provider may manage a reaction by slowing the infusion rate or treating with medicine such as antihistamines, fever-reducing agents and/or corticosteroids or possibly stopping the medication and then restarting with a longer infusion time. For patients who have had symptoms of hypersensitivity reaction to enzyme replacement therapy, the doctor may consider treating the patient with antihistamines and/or corticosteroids before an infusion to help prevent such a reaction from happening.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Side effects more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

The side effect profile in elderly patients was generally similar to that seen in pediatric and other adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering other existing medical conditions.

As with all therapeutic proteins, there is a potential for developing antibodies to VPRIV. In clinical studies, 1 of 54 (2%) patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. One additional patient developed antibodies to VPRIV during an extension study. It is unknown if having antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

For additional safety information, please click here for Full Prescribing Information including WARNING for Risk of Anaphylaxis, and discuss with your doctor

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.

For more information, contact Takeda at 1-877-TAKEDA-7 (1-877-825-3327), or by email at medinfous@takeda.com