12-year-old Jack Bolton has had spinal muscular atrophy (SMA) since he was born. He is confined to his wheelchair because of SMA, which causes his muscles to weaken as he…
I want you to think about spinal muscular atrophy (SMA), a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Then, think about the word: DIGNITY. Dignity…
Did you know that the #1 genetic cause of death for infants is spinal muscular atrophy (SMA)? It quickly robs the infant of physical strength, rendering the child unable to…
Who could be against the health and well-being of newborn babies? Surely, not any decent human being. Babies come into this world in an incredibly vulnerable state. They have essentially…
2017 Muscular Dystrophy Association Scientific Conference was a beacon of light! Researchers from academic medical centers, pharmaceutical companies, children’s’ medical centers, biotech, engineering, and veterinary medicine came together to share…
An Australian couple shares their story of finding out that their daughter Aviana, has spinal muscular atrophy (SMA). SMA is a rare genetic disease, which effects the nerves that control…
Treading water is difficult. Sometimes, that’s how it feels navigating a new diagnosis. It’s like trying to keep your head above water in a vast, foreign ocean, as waves of information…
Sometimes, the race to develop breakthrough treatments for rare and devastating illnesses can feel less like the Indy 500 and more like a game of Curling played by armless sloths.…
Genetic testing for rare diseases such as spinal muscular atrophy (SMA) before you conceive can be an important step in starting or building a family. It can be vital to…
An 8-year-old child in Fresno, California is receiving an FDA approved miracle drug that will possibly lengthen her life. Hayden Calafiore is the first to receive the injection as a…
Cure SMA is a nonprofit organization that has officially launched their 2017 SMA survey. This is a survey is available to those with spinal muscular atrophy (SMA) across the world. Also,…
The idea that a possibly life-altering medication is being denied to children is certainly something that will get the fires of the masses stoked. Imagine how inflamed the mob will…
Welcome back to Editor's Choice Patient Worthians! This week, a PW Contributor told a love story about how her husband's bipolar disorder helps her manage her rare disease. We also have an…
Imagine walking through the woods at night. You have no lantern and no flashlight—not even a match. The darkness is suffocating, and you’re terrified. But you keep blindly stumbling. It’s…
A frequent problem with for rare disease patients worldwide is being correctly diagnosed. Prior to the diagnosis you won't know the proper treatments, you don't know why you're experiencing your…
The first medication to combat spinal muscular atrophy (SMA) has recently been approved by the United States Food and Drug Administration. It is approved for use by children as well…
Genetic testing for Spinal Muscular Atrophy (SMA). Most US-born newborns are undergo testing to find out if they have certain genetic conditions. These tests vary from state to state. There…
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