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Choosing a Specialty Pharmacy to Support Hereditary ATTR Amyloidosis Patients

Hereditary ATTR amyloidosis (hATTR) is a rare, debilitating genetic condition affecting approximately 50,000 people worldwide. hATTR is characterized by the buildup of abnormal deposits of protein fibers called amyloid in the body’s organs and tissues, interfering with their normal functioning. ​These protein deposits most frequently occur in the peripheral nervous system, resulting in a loss of sensation, pain, or immobility in the arms, legs, hands, and feet. Amyloid deposits can also affect the functioning of the heart, kidneys, eyes, and gastrointestinal tract. hATTR amyloidosis is a genetically transmitted disease and can affect 50% of family members. This disease typically occurs in adults ranging from 30 to 70 years of age, depending on the mutation. (Source: and

hATTR amyloidosis is a physically debilitating disease that can contribute significantly to morbidity and a decline in quality of life, negatively affecting activities of daily living. The rate at which hATTR amyloidosis progresses is unpredictable but left untreated, death can occur within a few years of initial clinical presentation. (Source:

The signs and symptoms of hereditary ATTR amyloidosis (hATTR) are numerous and can affect many major organs. Although some mutations are associated predominantly with polyneuropathy or cardiomyopathy, most patients with hATTR amyloidosis have mixed clinical phenotypes, including neurologic and cardiac symptoms. Because the symptoms of hATTR amyloidosis do not always start in one specific organ, the systemic nature of the disease is often masked. As a result, it is vital to look for a cluster of red-flag symptoms including, but not limited to, cardiovascular manifestations, debilitating and uncontrolled GI symptoms, autonomic conditions, unexplained weight loss, and more. (Source:

When choosing a specialty pharmacy to support Hereditary ATTR amyloidosis (hATTR) patients, consider a team experienced in rare diseases, ready to listen, provide counseling on treatment, help with side-effect management, and assist with sourcing third-party financial assistance. As part of the patient’s treatment, Orsini Specialty Pharmacy provides the following:

Access to hATTR MedicationsChoose a specialty pharmacy with access to hATTR medications. Orsini Specialty Pharmacy has access to the following drugs:

  • Onpattro (Intravenous infusion)
  • Vyndamax (Oral)
  • Vyndaqel (Oral)

hATTR Care Team: Patients need an experienced hATTR Care Team who provides information and support throughout their journey and support for them and their caregivers.

In-Home Patient and Family-Centric Care: Look for a specialty pharmacy that provides infusion nursing services. Orsini Specialty Pharmacy utilizes a nationwide nursing network, enabling patients and their families to receive care at home. With the ability to individually match our nurses with the skill and care level required by each patient, we create a unique tailored high-touch opportunity to optimize their care plans and clinical outcomes.

Shipping & Coordination of Care: Patients receive free priority overnight delivery of medications and necessary medical supplies. Orsini manages care coordination between prescribers, care sites, nurses, and patients.

Financial Assistance: hATTR medications are costly, and many patients cannot afford the co-pays associated with their health insurance. Orsini helps eligible patients enroll in manufacturer co-pay and foundation support programs upon request.

If you are a prescriber or physician office representative, click here to access hATTR enrollment forms. In addition, you can always connect with your Orsini rep to schedule a call and answer any questions. If you are a prospective patient, click here to start your patient enrollment form.