Spinal muscular atrophy is both rare and deadly. It’s uncommon enough that most people never worry about it-- but the consequences that happen if it goes undetected are devastating. That’s…
Meisyn Ellison, a 13-year-old in Utah, adopted from China, battles the debilitating effects of her rare disease and now seeks to find her biological parents in hopes to give doctors…
The Lewis family has lived through it all. Their first born was diagnosed with spinal muscular atrophy (SMA) and did not live long, having passed away in 2013, reports Deseret News. And…
Little Natalie Essex was diagnosed with spinal muscular atrophy type 2 (SMA) when she was only a year and a half, reports CBC News. It all started when her mother, Meredith,…
Still getting over that crazy Super Bowl game?! If you're an Eagles or Patriots fan, you might need abit more time to gloat/recover - but for the rest of us,…
There is new hope for patients and their families suffering from spinal muscular atrophy (SMA) reported AOL UK News. SMA is a physically debilitating and aggressive disease, and now a new…
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 change forever. She was diagnosed with…
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 rare genetic disorder called…
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 is the cost of life?…
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 it? That's what a group of…
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 to treat spinal muscular atrophy (SMA).…
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, and the smaller the patient population,…
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 ameliorate symptoms of a mild form…
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 of the results? The police…
Milestones. Oh, the firsts! When you have a new baby, your whole life revolves around firsts: first bath, first smile, first tooth, first roll, first uninterrupted night of sleep (yes!).…
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 eyes, and breathe it in. Sometimes, inspiration just…
The United States' Orphan Drug Act (ODA) was enacted in 1983. Years later, it has failed to fuel the creation of orphan drugs to treat rare diseases, including spinal muscular…
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 parent wants for their child.…
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 atrophy (SMA) since she was…
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 that the Muscular Dystrophy Association…
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 six weeks old before he…
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 the increasingly complex health insurance…
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 number, letter, color, or object…
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 suit was inspiring the next…
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 is the first and only…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
