Tag: Spinal Muscular Atrophy (SMA)

Young Adults, MDA Ambassadors, News

Oh, Hello 2017 — I’m Ready for You

Joe Akmakjian

When I was asked to continue in my role as National Ambassador for MDA in 2017, I had to think about it for about a nanosecond before I gave my answer. Yes, of course! So it’s official: I’ve re-upped for another tour. 2016 will be a hard act to follow, but now that I’m a registered frequent flyer and have a year of ambassadorship under my belt, I can’t wait to try to top it. In fact, I’ve taken the liberty of compiling a list of experiences I’m looking forward to in 2017. Join me, won’t you?

Read More
Research, Uncategorized

Encouraging Results in Nusinersen (Spinraza) Trial for Type 2 SMA

Amy Madsen

In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging interim results from the late-stage CHERISH clinical trial to test the experimental drug nusinersen (brand name Spinraza) in children with type 2 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label . . .

Read More
Research, Uncategorized

Biogen and Ionis Pharmaceuticals: A Message to the SMA Community

Amy Madsen

In a joint statement today, Biogen and Ionis Pharmaceuticals announced encouraging results from the late-stage CHERISH clinical trial to test the experimental drug nusinersen (brand name Spinraza) in children with type 2 spinal muscular atrophy (SMA). Based on the results, investigators have stopped the trial to allow all participants to transition into an open-label trial . . .

Read More
Uncategorized

Scarlett’s Night at MDA Muscle Team Gala

MDA Admin

Hockey season is finally underway, and the Chicago Blackhawks have added one more fan to their roster. No, this fan is not a burly teenager who loves to play hockey, or someone mesmerized by the Blackhawks and their Stanley Cup glory of the past six years. This fan is 5-year-old Scarlett Sepe, an adorably outgoing . . .

Read More
Research, Uncategorized

FDA Will Review Nusinersen for Treatment of SMA

Amy Madsen

The U.S. Food and Drug Administration (FDA) has accepted Biogen’s New Drug Application for nusinersen (brand name Spinraza) for the treatment of spinal muscular atrophy (SMA) and granted Priority Review. Priority Review status is reserved for drugs that offer significant improvements over existing options or provide a treatment for a disorder for which no approved . . .

Read More
Uncategorized

Cooper’s Journey with Spinal Muscular Atrophy

MDA Admin

A couple of years ago MDA got to know the Somers family… While Crystal and Cooper continue to mourn the loss of an incredible husband and father, they are able to focus on and appreciate the positives in their life now. Cooper recently turned 6, started first grade and is ready – and excited – . . .

Read More
News, Research

Biogen Submits New Drug Application for Nusinersen to Become an Approved Treatment for SMA

Amy Madsen

In a joint statement today, Biogen and Ionis Pharmaceuticals announced that Biogen has completed the rolling submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of nusinersen, an investigational treatment for spinal muscular atrophy (SMA). Submission of the NDA is the first step toward getting approval . . .

Read More
News, Advocacy

MDA Moves Newborn Screening Forward in Partnership with Cure SMA

Kristin Stephenson and Amanda Haidet-Phillips

This September as we recognize Newborn Screening Awareness month, we are proud to announce a new partnership with Cure SMA aimed at moving newborn screening forward for spinal muscular atrophy (SMA)—the leading genetic cause of death in infants. This is an exciting time for the neuromuscular disease community as decades of research are translating into . . .

Read More
First...5678...Last