Spinraza (nusinersen) has been approved by the FDA and is now available for treatment of patients with SMA due to SMN1 mutations. The CUMC - SMA Clinical Research Center was the first site in the world to administer Spinraza in a clinical trial.
Patients who meet the following criteria may be eligible for treatment with Spinraza:
- Patients who have genetically confirmed SMA and meet the medical criteria for treatment.
- Patients who are registered and receiving care with a Columbia University doctor.
- Patients who are able to follow-up with their Columbia University doctors after treatment.
- Patients who can be evaluated by their Columbia University Health Care Provider, including physicians and nurse practioners, six months after receiving their Spinraza treatment.
It is important for all those interested to know that not all patients will be eligible for treatment, that treatment could be discontinued if a patient does not respond to the drug, and that treatment services are dependent upon availability of resources.
For each patient who meets clinical criteria for eligibility, NYPH - CUMC will work with their payor to attempt to arrange financial coverage.
Patients who are covered by payors that are not contracted with NYPH - CUMC will need to either seek care at a provider within their payor’s network of participating providers or obtain out-of-network approval from their payor for coverage at NYPH - CUMC.
There are no clinical trial data regarding safety and efficacy of Spinraza in SMA patients over age 18. New York-Presbyterian and Columbia University Medical Center physicians are reviewing alternative mechanisms to administer Spinraza to adult SMA patients.
If you are interested in additional information or would like to make an appointment for an initial evaluation, please call Patient Care in the CUMC Department of Neurology, Division of Child Neurology at (646) 426-3876, then select option 5; please have your doctor fax referrals to (212) 342-2893 attention Nicole LaMarca, DNP, MSN, CPNP. Please have your doctor indicate that you are interested in Spinraza.
If you or your child has previously been seen at the Columbia University Neuromuscular and SMA Clinical Research Center but has not had a follow-up visit in the last 6 months, please schedule an appointment by calling (646) 426-3876, then select option 5 to speak with an agent.