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…
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…
Spinal muscular atrophy (SMA) is a devastating, rare inherited disorder that causes muscle weakness and degeneration. There are four types of spinal muscular atrophy and currently there is no known cure…
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 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.…
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…
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 inherited disorder that disables the…
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