Researchers at Michigan Medicine at the University of Michigan are seeking caregivers of children living with spinal muscular atrophy (SMA) to participate in a survey about treatment decision making. Over the last five years, three treatments have been approved and are changing life expectancy and physical functioning for children with SMA. The goal of this survey . . .
It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide to raise a little green (more than $323 million, to be exact) both to send kids to MDA Summer Camp and to fund groundbreaking research . . .
Feb. 28 is Rare Disease Day, when the collective rare disease community raises awareness of the conditions with which we live and advocates for access to new novel treatments like Spinraza, the first FDA-approved disease-modifying drug for spinal muscular atrophy (SMA), a rare neuromuscular disease that affects people like 18-year-old Marley Robinson. Spinraza is making . . .
Joining a growing number of companies developing robotic devices for those living with mobility impairments, Ontario’s Trexo Robotics just launched the newest in its line of robotic walking aids specifically for children, the Trexo Home. Unlike the company’s Trexo Plus, a set of wearable robotic legs that attach to a walker for use in clinical . . .
This week, Arkansas Gov. Asa Hutchinson signed into law a bill, HB 1074, that requires the state to add spinal muscular atrophy (SMA) to its newborn screening panel. SMA is the No. 1 genetic cause of death in infants, affecting approximately 1 in 10,000 babies. MDA was proud to advocate for the addition of SMA . . .
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 . . .