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…
Earlier this year, Patient Worthy featured Ashley and her non-profit Love Not Lost – an organization that provides portrait sessions and photo albums for families dealing with a terminal illness…
Most disabilities in Pakistan are caused by three diseases: Spinal Muscular Atrophy, Spinal Cord Injury, and stroke. With this in mind, a group in the country’s capital has launched the…
The gene therapy development company, AveXis, Incorporation, just announced that they will be expanding their clinical trial development program for the potential gene therapy treatment: AVXS-101, reported Globe News Wire.…
According to a story from financialbuzz.com, the pharmaceutical company AveXis, Inc., is planning to begin preparations to submit a Biologics License Application (BLA) to the Food and Drug Administration for…
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…
An Ottowa family is overjoyed after some good news. They have just been informed that their insurance company is offering to cover $120,000 worth of treatment to cover their son's…
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 rare disease patient is getting the recognition she deserves for her fearless outlook on death and her refusal to submit to affliction. On Sunday, the winners were announced for…
While other kids his age run amok in the wildness of their Terrible Two's, Lincoln Woodmass remains immobile. No running, no jumping and the possibility of never walking again. You…
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,…
Rare diseases like ALS and SMA cause damage to motor neurons. Often, these conditions lead to death, in part because muscles in the chest are wasted away and the patient…
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…
In recognition of Spinal Muscular Atrophy Awareness Month, Joe Akmakjian, an SMA warrior/blogger, is promoting a unique understanding of his rare disedsase. Originally published on the Muscular Dystrophy Association's Strongly…
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!).…
One of my favorite places to visit is Savannah, Georgia. And what better reason to visit than an spinal muscular atrophy (SMA) charity event? Who: SMA Angels What: SMA Angels Charity Ball…
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.…
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