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About the Disease

FABRY DISEASE

About the Disease

About our Patient Care

Fabry disease belongs to a group of diseases known as lysosomal storage disorders. It is a rare inherited disorder of lipid (fat) metabolism which results from the deficient activity of the enzyme, alpha-galactosidase A (a-Gal A). This leads to a buildup of a particular type of fat in the body's cells, called globotriaosylceramide or GL-3, that affects many parts of the body. Signs and symptoms may include episodes of pain, particularly in the hands and feet, clusters of small, dark red spots on the skin, a decreased ability to sweat, cloudiness of the front part of the eye and hearing loss. Complications such as progressive kidney damage, heart attack, and stroke can be life-threatening.

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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.

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As part of treatment, we provide:

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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.

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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.

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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.

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Financial Assistance

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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.

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  • Berinert (C1 esterase inhibitor)
    Brand Name | Berinert Manufacturer | CSL Berhing Route of Administration | Intravenous (IV) injection Approved Indication | Treatment of acute abdominal, facial, or laryngeal HAE attacks in adult and pediatric patients Self-Administration | Yes, after training Product Website | www.berinert.com
  • Cinryze (C1 esterase inhibitor)
    Brand Name | Cinryze Manufacturer | Shire Route of Administration | Intravenous (IV) injection Approved Indication | Routine prophylaxis against angioedema attacks in patients with HAE Self-Administration | Yes, after training Product Website | www.cinryze.com
  • Firazyr (icatibant)
    Brand Name | Firazyr Manufacturer | Shire Route of Administration | Subcutaneous (SC) injection Approved Indication | Treatment of acute attacks of HAE in adults 18 years of age and older Self-Administration | Yes, after training Product Website | www.firazyr.com
  • Haegarda (C1 esterase inhibitor)
    Brand Name | Haegarda Manufacturer | CSL Behring Route of Administration | Subcutaneous Approved Indication | For routine prophylaxis to prevent HAE attacks in adolescent and adult patients. Self-Administration | Yes Product Website | www.haegarda.com
  • Kalbitor (ecallantide)
    Brand Name | Kalbitor Manufacturer | Dyax Corp Route of Administration | Subcutaneous injection Approved Indication | For routine prophylaxis to prevent HAE attacks in adolescent and adult patients. Self-Administration | No Product Website | www.kalbitor.com
  • Ruconest (C1 esterase inhibitor)
    Brand Name | Rocunest Manufacturer | Pharming N.V. Route of Administration | Intravenous (IV) injection Approved Indication | Treatment of acute attacks of HAE in adult and adolescent patients. Self-Administration | Yes, after training Product Website | www.ruconest.com
  • Takhzyro (lanadelumab-flyo)
    Brand Name | Takhzyro Manufacturer | Shire Route of Administration | Subcutaneous injection Approved Indication | Prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older Self-Administration | Yes, after training Product Website | www.takhzyro.com
Supported Treatments

Supported Treatments

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.

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Partnering with Prescribers

Partnering with Prescribers

Licensed prescribers can submit a completed enrollment form directly to our Admission team. We will begin the process and communicate with your office every step of the way.

Send Us A Script

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