AveXis, a part of Novartis, is working “diligently” with the U.S. Food and Drug Administration to identify steps…
Articles by Grace Frank
The U.S. Food and Drug Administration (FDA) has placed a partial hold on a Phase 1/2 clinical trial testing…
AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation…
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a…
Screening newborns for genetic diseases for which treatments exist that can prevent crippling or deadly progression, especially for rare…
When she was born in London on Sept. 9, 2018, Tora Patgiri showed no signs of the spinal muscular atrophy…
[Editor’s Note: In the conclusion of this two-part interview with the Lackey family of Arizona about their daughter’s treatment…
[Editor’s Note: This is the first of a two-part series on the Lackey family of Arizona, whose daughter Stella became…
Within a lifetime, spinal muscular atrophy has leapt from a disease “nobody knew anything about” to a treatable —…
How Zolgensma (onasemnogene abeparvovec-xioi), the gene therapy once known as AVXS-101, came into being is a tale fairly common to…