Tags Posts tagged with "SMA"

SMA

Skylar Marie came into the world as a beautiful healthy baby. It wasn't until 2 months later that her and her family's world would...

Glenda Thompson is a proud grandmother who will stop at nothing to ensure her grandson lives a comfortable life. 4-year-old Cole Thompson is suffering from...

A Futurism article this week put into very human terms the ongoing question we face when it comes to rare disease treatment and Pharma: What...

Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality in the USA. So why aren't hospitals in Texas screening newborns for...

Just days before Christmas 2016, the FDA gave a present to some of the children in the United States—they approved the first treatment ever...

I don’t think it comes as a shock to anyone to say the costs of drugs are too damn high. The rarer the disease,...

In a study supported by the National Institute of Health (NIH), researchers at Iowa State University found that a drug called A15/283 helped to...

Whenever I feel the stress of life getting to me, I hop over to YouTube and search “random acts of kindness” or “heartwarming acts.” Some...

Milestones. Oh, the firsts! When you have a new baby, your whole life revolves around firsts: first bath, first smile, first tooth, first roll,...

Inspiration is everywhere—from the words of your favorite writer to the blooms and leaves on your morning walk. You just need to open your...

The United States' Orphan Drug Act (ODA) was enacted in 1983. Years later, it has failed to fuel the creation of orphan drugs to...

All Camden Fuller’s parents want is for him to have the chance to be healthy, grow up, and live a fulfilling life. That’s what every...

Over ten years ago, four close friends founded a charity called Angie’s Hope. One of the founders, Angie Lee, has been living with spinal muscular...

According to Seacoast Online early this year, Cohl Capparelli was diagnosed with spinal muscular atrophy (SMA) about two years ago. SMA is one of the diseases...

Kumail was diagnosed with spinal muscular atrophy (SMA) and started Spinraza in an international trial. As The Herald Sun reports, he began the treatment at...

Some good news for patients with Spinal Muscular Atrophy (SMA) and their families! There's a new booklet out with detailed information on how to navigate...

Kids with spinal muscular atrophy (SMA) have a hard time with an evaluation method known as "pair-matching tests." They have a harder time identifying what...

When Ellen Ripley used the Caterpillar P-5000 Work Loader to pick up the xenomorph queen in the 1986 science-fiction masterpiece Aliens, who would've known...

The U.S. Food and Drug Administration (FDA) approved Biogen’s SPINRAZA™ (nusinersen) under Priority Review for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. SPINRAZA...

Spinal muscular atrophy (SMA) occurs when the immune system attacks motor neurons, resulting in the muscles surrounding the spinal cord atrophying (degenerating). SMA is an...

The line between genius and madness is often closer than people realize. Don’t tell Dr. Sergio Canavero that he can’t help his patient with...

Adyn’s Dream is a charity in Athens, Ohio. The organization provides live events that benefit families and their children that have received a diagnosis of...

On a beautiful summer day, a baby girl named Remi made her debut in the world. To all appearances, everything was normal—10 fingers, 10 toes,...

Passion. Passion doesn’t persist. It is there, and then it is gone. Standing on the diving board is pre-passion. Falling toward the water is...

Alexander Davis is from Tipton, Indiana. He has been living with spinal muscular atrophy (SMA). SMA prevents him from moving much and he is unable to...