Spinal muscular atrophy (SMA) is a genetic disease that affects the central nervous system, peripheral nervous system, and voluntary muscle movement, also known as skeletal muscle. A majority of the nerve cells that control muscles are located in the spinal cord, and when the patient’s muscles do not receive signals from those nerve cells, this results in their muscles getting smaller. The cause of spinal muscular atrophy is due to a missing gene that encodes the survival motor neuron (SMN) protein, which is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons).


SMA occurs in nearly 1 in 11,000 births in the United States. The age when SMA symptoms begin tends to correlate with the severity to which motor function is affected. For example, the earlier the age of onset, the greater the impact on the patient’s motor function. Children who display symptoms at birth or in infancy typically have the lowest level of functioning (Type 1). Later-onset SMA patients with a less severe type (Types 2 and 3, and in teens or adults, Type 4) generally tend to have higher levels of motor function. Newborn screening now includes SMA, which enables physicians to identify patients early on.

About our Patient Care

We at Orsini understand the sense of urgency in getting patients their medication as quickly as possible. As part of the patient’s treatment, we provide the following services:

Specialized Gene Therapy Team

We provide prescribers and patients with a dedicated single point of contact who manages care coordination between prescribers, sites of care, nurses, and patient caregivers, supported by an empathetic and experienced SMA Care Team.

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Quick Turn-Around Time

Orsini has an average turnaround time of 3 days from receipt of referral to delivery at site of care. We 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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Broad Payor Coverage

Orsini is contracted with more than 525 National Health Plans and PBM’s, and is a licensed Medicaid provider throughout the United States and its territories.

Prior Authorization and Reimbursement

Through extensive experience in reimbursement on both the medical and pharmacy side, we can help facilitate the prior authorization and reimbursement to ensure eligible patients obtain coverage for their therapy.

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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Supported Treatments

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