According to a story from GlobeNewswire, The pharmaceutical company AveXis, Inc., a company that specializes in the development of gene therapies, recently announced that its candidate AVXS-101 was awarded SAKIGAKE…
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…
According to a story from Charcot-Marie-Tooth Disease News, the U.S. Food and Drug Administration (FDA), under the leadership of commissioner Scott Gottlieb, has begun to change clinical trials by accepting…
The day after Christmas in 2016 brought a post-Holiday surprise to many in the U.S. with spinal muscular atrophy. December 26, 2016 marked the day that Spinraza was approved by…
According to a story from macleans.ca, eight-year-old Mason Stewart has been dealing with the effects of spinal muscular atrophy since he began to walk; he developed an unusual walking gait…
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…
One of the most impressive moments of childhood is when a young child takes his or her first steps. Natalie Essex, age four, loves to walk. For Natalie, spinal muscular…
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…
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