Check Out Cure SMA’s 2024 Annual State of SMA Report Cure SMA is pleased to announce the launch of our next State of SMA report, showcasing data through 2024. The annual data report highlights data from Cure SMA’s three databases: a patient-reported database with data from over 11,000 impacted individuals worldwide that also incorporates longitudinal […]
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Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review
One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). This helps the FDA make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as a variety of new “add-on” therapies and
