AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation issues” that led to inaccuracies in its application to the U.S. Food and Drug Administration prior to the treatment’s approval, but failed to inform regulators until more than a month…
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SMA News Today, having launched forums and added regular columnists, is now running a month-long series to coincide with Spinal Muscular Atrophy Awareness (SMA) Month that highlights the challenges and perseverance of patients and caregivers. Called “31 Days of SMA” and running through August, the series spotlights a person each…
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…
Severe brain abnormalities are evident in children born spinal muscular atrophy type 0, also known as prenatal onset SMA, who survive into early childhood, according to a recent case report. The study, “Severe brain involvement in 5q spinal muscular atrophy type 0,” was published in the Annals of…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
Screening newborns for genetic diseases for which treatments exist that can prevent crippling or deadly progression, especially for rare disorders like spinal muscular atrophy, has a way to go in the United States. Only seven states require this disease to be among the dozens tested from a blood sample…
When she was born in London on Sept. 9, 2018, Tora Patgiri showed no signs of the spinal muscular atrophy (SMA) type 2 she would be officially diagnosed with some eight months later in Ohio. The baby ate well, waved her arms easily, and grasped objects at will. Until a…
Clinicians have described the case of an Iranian teenager with a rare type of spinal muscular atrophy (SMA) associated with progressive myoclonic epilepsy (PME). The case report study, “A novel case report of spinal muscular atrophy with progressive myoclonic epilepsy from Iran,” was published in the…
The European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv, a potential add-on therapy for patients with spinal muscular atrophy (SMA). Reldesemtiv, developed by Cytokinetics and Astellas, is meant to complement existing SMA treatments. It is a fast skeletal muscle troponin activator…
A new long-term partnership with Catalent Biologics will allow Zolgensma’s developer AveXis to increase production of the gene therapy and gain access to further technical expertise. Zolgensma was approved by the U.S. Food and Drug Administration (FDA) to treat pre-symptomatic newborns through 2-year-olds with any type…
