Spinal muscular atrophy (SMA) is a rare, genetic and progressive neuromuscular disorder characterized by weakness of the skeletal and respiratory muscles. SMA is caused by a missing gene that encodes the survival motor neuron (SMN) protein. The SMN protein is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons).
Approximately 10,000 people in the United States have SMA, which occurs in nearly 1 in 11,000 infants and is the number one genetic cause of infant death. The disease can affect any race, age or gender.
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.
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.
Insurance and Financial Assistance
We coordinate between the health insurance provider and patient/caregiver to ensure the prescribed therapy is approved. We work with site-of care facilities and other funding entities to help facilitate obtaining approval for the prescribed therapy.
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.
Zolgensma (onasemnogene abeparvovec-xioi)
Brand Name | Zolgensma
Manufacturer | AveXis
Route of Administration | Intravenous (IV) infusion
Approved Indication | Treatment of pediatric patients less than 2 years of age with SMA
Self-Administration | No
Product Website | www.zolgensma.com