Jenni Gold has been writing, directing and producing film shorts, movies and TV shows for more than 20 years. She’ll tell you, since she was a kid making movies with a Hi8 camcorder, she’s been in love with Hollywood for a lot longer. She’ll also tell you there’s a problem with it. It’s failing the . . .
Today, Novartis, the parent company of AveXis Inc., announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) under Priority Review for Zolgensma (formerly known as AVXS-101), a gene therapy for the treatment of spinal muscular atrophy (SMA) type 1. Priority Review status requires the FDA to review . . .
This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. AveXis is also submitting regulatory applications for the therapy in Europe and Japan. If approved, AVXS-101 would . . .
On Oct. 3, Genentech announced interim results from the dose-finding parts of the pivotal FIREFISH and SUNFISH studies investigating risdiplam (RG7916) in spinal muscular atrophy (SMA). Risdiplam is an investigational SMN2-splicing modifier that is given by mouth (or g-tube) and distributes widely throughout the body. Risdiplam is designed to help the SMN2 gene produce more . . .
This is an unprecedented time for patients living with spinal muscular atrophy (SMA). It has now been almost two years since Spinraza became the first FDA-approved treatment for SMA, and in that time it’s proven to be a transformative therapy. Although there is much more work to be done, Spinraza has made a significant impact . . .
After significant collaborative work among many stakeholders in the spinal muscular atrophy (SMA) community, we are pleased to share that the Secretary of Health and Human Services (HHS), Dr. Alex Azar, has accepted the recommendation to add SMA to the Recommended Uniform Screening Panel (RUSP) for newborns. This is a landmark decision for the SMA . . .
The Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), a federal committee that oversees newborn screening, voted today to add spinal muscular atrophy (SMA) to the national list of disorders recommended for screening at birth. Following today’s vote, the Secretary of Health and Human Services (HHS) will make a final decision on whether . . .
Kevan Chandler, who lives with SMA and traveled through Europe in 2016 in a backpack, is heading to China. Along the way, he hopes to inspire others and “demonstrate that life is full of possibility and hope.”