Offered by: Nemours Children's
Location: Orlando, Fla.
International SMA Consortium Spinal Muscular Atrophy Patient Registry (iSMAC SMA Registry)
SMAC is an ongoing data collection effort for Spinal Muscular Atrophy, or “SMA”. The main purpose of this research study is to learn more about the disease and the response to treatment. We hope that by collecting and analyzing this information will improve care of those with SMA.
All patients with SMA who receive care at Nemours Children’s Hospital, Florida are eligible to participate in this natural history registry. Other patients with SMA at 4 other sites in the United States and at sites in the United Kingdom and Italy will also participate in this Registry.
There are no additional procedures, clinic visits or expectations from you as a result of participation in the iSMAC SMA Patient Registry. By agreeing to be in the Registry, you allow a staff member from our SMA clinic to review your medical records and enter information into an electronic form that is sent to the University of Rochester that is analyzing and storing data. This de-identified data will be sent electronically throughout the year to the sponsor of the registry — Biogen.
A summary of your child’s health (height, weight, genetic test results and physical therapy assessments) and treatments (medications and other therapies) will be submitted electronically through the Electronic Medical Records System. There are no experimental procedures that are part of this study. De-identified information will be collected and shared with the sponsor of the study and other centers. There are two types of visits that your child will have to undergo as a part of standard treatment. Baseline visit (initial visit) and the follow- up visits one to four times a year. All the procedures that are performed at clinic visits are part of the standard treatment that your doctor schedules for you.
Data will be collected as a result of the following standard treatment procedures:
Trial Name: International SMA Consortium Spinal Muscular Atrophy Patient Registry (iSMAC SMA Registry)
IRB#: 1238325
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