Cause of Inflammation That Led to STRONG Trial Hold of AVXS-101 ‘Unknown,’ AveXis Says
AveXis, a part of Novartis, is working “diligently” with the U.S. Food and Drug Administration to identify steps…
Articles by Grace Frank
FDA Places Partial Hold on STRONG Trial of AVXS-101, Citing Concern Seen in Animal Study
The U.S. Food and Drug Administration (FDA) has placed a partial hold on a Phase 1/2 clinical trial testing…
AveXis Knew of ‘Manipulated’ Data in Zolgensma Application and Failed to Inform FDA, Agency Says
AveXis, the company that developed the gene therapy Zolgensma to treat spinal muscular atrophy (SMA), knew of “data manipulation…
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a…
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
Screening newborns for genetic diseases for which treatments exist that can prevent crippling or deadly progression, especially for rare…
‘Small Differences’ That Mark SMA Type 2 in Baby Girl Bring Family from UK to Ohio for Zolgensma Treatment
When she was born in London on Sept. 9, 2018, Tora Patgiri showed no signs of the spinal muscular atrophy…
Stella’s ‘Very Comfortable’ Hour with Zolgensma and Pleas for Newborn Screening: The Lackeys on Their SMA Journey
[Editor’s Note: In the conclusion of this two-part interview with the Lackey family of Arizona about their daughter’s treatment…
From Parents’ Nightmare to Cause for Hope: The Lackey Family on Learning of SMA and Trying Zolgensma
[Editor’s Note: This is the first of a two-part series on the Lackey family of Arizona, whose daughter Stella became…
‘Excited for Next Generation,’ People Living with SMA Say of Leap in Treatment Options
Within a lifetime, spinal muscular atrophy has leapt from a disease “nobody knew anything about” to a treatable —…
Zolgensma’s Journey from Lab Idea to Gene Therapy for SMA
How Zolgensma (onasemnogene abeparvovec-xioi), the gene therapy once known as AVXS-101, came into being is a tale fairly common to…