A single injection of Zolgensma into the spinal canal (intrathecal) can provide a clinically meaningful response in motor function gains among young children, 2 to 5 years old, who have spinal muscular atrophy (SMA) type 2, recent trial data show. Half of these older patients…
In an unprecedented decision reached Sept. 26 on the eve of Rosh Hashana — the Jewish New Year — Israel’s top health insurer, Clalit, said it would cover the $4.25 million cost of one-time Zolgensma gene therapy for two Israeli babies born with spinal muscular atrophy (SMA).
Babies with spinal muscular atrophy (SMA) treated with Zolgensma before experiencing symptoms continue to reach motor milestones in a normal timeframe, according to updated Phase 3 results. “With genetic testing in hand and presymptomatic treatment, we can look forward to a future in which age-appropriate motor milestone development…
With increasing insurance coverage and AveXis’ plans to broaden access to older spinal muscular atrophy (SMA) patients, the U.S. Food and Drug Administration (FDA) remains “very confident” in the use of Zolgensma despite preclinical data concerns, according to an official from the agency. Cure SMA…
Skye, a nine-month-old baby with spinal muscular atrophy (SMA) type 1, is reaching new milestones every week and is “the happiest child” after being treated with Zolgensma, according to her mother Kasia. “She is almost sitting, her head and neck are really strong, and she is reaching above…
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…
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…
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…
[Editor’s Note: In the conclusion of this two-part interview with the Lackey family of Arizona about their daughter’s treatment with Zolgensma at 6 weeks old, we look at treatment day, advice they have to make it easier, and the family’s decision to advocate for screening newborns for SMA.] Samantha…
[Editor’s Note: This is the first of a two-part series on the Lackey family of Arizona, whose daughter Stella became the first baby west of Missouri to be treated outside of a clinical trial with Zolgensma on July 3. Here, the family talks about how their daughter came to be…