GAUCHER DISEASE TYPE 1 TREATMENT
About the Disease
Gaucher disease type 1 is the most common type form of Gaucher disease, a lipid metabolism disorder, that causes the liver and spleen to become enlarged, bone disease and, sometimes, lung and kidney problems. It does not affect the brain, as seen in type 2 and type 3 Gaucher disease. While there is no cure, type 1 can be effectively treated with enzyme replacement therapy (ERT).
About our Patient Care
Our dedicated Care Team will work with the physician's office and insurance on a patient's behalf so that our patients have more time to focus on their treatment and quality of life. We are here to listen, provide
counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.
As part of treatment, we provide:
Dedicated Care Team
We provide prescribers and patients with a dedicated care team for each of our disease state programs. This includes a primary pharmacy contact, clinical education, side-effects management, scheduled refill calls, and a direct phone, fax and email address.
In-Home Patient- and Family-Centric Care
We administer a nationwide nursing network, allowing patients to pursue in-home treatment or receive training on self-administrated drugs. Our nurses are ideally positioned to establish a partnership with patients and their families in order to provide clinical education and coaching that strengthen the patient's capacity to achieve therapy goals.
Shipping and Coordination of Care
We provide free overnight delivery of medications and necessary medical supplies and manage the coordination of care between prescribers, sites of care, nurses and patients to ensure dispenses and drug delivery align with the date and location of service.
Financial Assistance
We successfully identify and assist enrolling patients into any and all manufacturer co-pay and foundation support programs in order to minimize a potential financial burden to patients given the high-cost of specialty medications.
24/7/365 Access to a Pharmacist
We provide prescribers and patients access to a pharmacist 24/7/365 to ensure patients are always cared for.
Supported Treatments
Click on the specialty drug name below to expand and see more information.
Cerdelga (eliglustat)
Brand Name | Cerdelga
Manufacturer | Genzyme
Route of Administration | Oral
Approved Indication | Gaucher Disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test
Self-Administration | Yes
Product Website | www.cerdelga.com
Cerezyme (imiglucerase)
Brand Name | Cerezyme
Manufacturer | Genzyme
Route of Administration | Intravenous (IV) infusion
Approved Indication | Type 1 Gaucher disease; associated with one or more of the following: anemia, thrombocytopenia, bone disease, hepatomegaly, splenomegaly
Self-Administration | Yes, after training
Product Website | www.cerezyme.com
Elelyso (taliglucerase alfa)
Brand Name | Elelyso
Manufacturer | Pfizer
Route of Administration | Intravenous (IV) infusion
Approved Indication | Treatment of patients with a confirmed diagnosis of Type 1 Gaucher disease
Self-Administration | No
Product Website | www.elelyso.com
Miglustat
Brand Name | Miglustat
Manufacturer | Edenbridge Pharmaceuticals, LLC
Route of Administration | Oral
Approved Indication | For the treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.
Self-Administration | Yes
VPRIV (velaglucerase-alfa)
Brand Name | VPRIV
Manufacturer | Shire
Route of Administration | Intravenous (IV) Infusion
Approved Indication | Long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease
Self-Administration | No
Product Website | www.vpriv.com