Zolgensma, approved for children with spinal muscular atrophy (SMA) up to 2 years old, has two claims to fame: It is both the first gene therapy ever developed to treat a neuromuscular disease, and it’s by far the world’s most expensive drug. At $2.125 million, a 60-minute intravenous…
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From its very first clinical trial — using a dose widely frowned upon — Zolgensma has had a “transformative” impact on spinal muscular atrophy (SMA) and on gene therapy as a whole, said Jerry Mendell, MD, a principal investigator across Zolgensma’s clinical program. “We did our best, I think, to make…
How Zolgensma (onasemnogene abeparvovec-xioi), the gene therapy once known as AVXS-101, came into being is a tale fairly common to basic science: An idea that progressed from cell work in the lab to experiments with animals and, ultimately, testing as a possible treatment in patients. Where Zolgensma’s story diverges from…
RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
“Great news!” was the reaction of one SMA News Today columnist to word of a first gene therapy, Zolgensma, being approved to treat infants and children up to age 2 with spinal muscular atrophy, while others on the site’s SMA community forum called it “an amazing advancement” and “such a…
Zolgensma (onasemnogene abeparvovec-xioi), the newly approved gene therapy to treat SMA in infants and children under 2 years old, works by delivering directly to motor neurons a healthy copy of the SMN1 gene that is damaged by mutations in these patients and unable to make an essential protein. Carried…
Bogdana Patrascu will never forget the day she learned that her then-3-year-old daughter, Alberta, had spinal muscular atrophy (SMA). The news came in a March 2016 phone call from Alberta’s pediatric neurologist, as Patrascu — a radiologist in Timisoara, Romania — was driving home from work. She had taken…
Zolgensma, a first gene therapy for spinal muscular atrophy — and first for any chronic neurologic disease — is now an approved and potential “one-time” intravenous treatment for pre-symptomatic newborns through 2-year-olds with any type of SMA, the U.S. Food and Drug Administration (FDA) announced today, issuing an historic decision.
A child who barely moved in the womb in the final months before his birth and then had multiple bone fractures at birth was diagnosed with spinal muscular atrophy (SMA) type 0, researchers reported. The case study, “Type (0) spinal muscular atrophy associated with fractures at birth,” was…
Treatment with the experimental oral therapy risdiplam has been enabling patients with spinal muscular atrophy (SMA) to achieve key development milestones and clinically meaningful motor improvements beyond the natural history of the disease. Susan Begelman, MD, vice president, Neurology, Nonmalignant Hematology, Influenza, U.S. Medical Affairs at Genentech,…
