According to a story from physiciansweekly.com, a recent report from the Institute for Clinical and Economic Review (ICER) suggests that, pending more data about overall price and longterm success rates,…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
Who's in the mood to help grant a young girl her Christmas wish this year?! People who know 10-year-old Paola Vega describe her as a cute, spunky princess who can't…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
According to a story from MarketWatch, Annie Wilson was diagnosed with spinal muscular atrophy when she was only six months old. With no real treatment options available, doctors told her…
According to a story from kentlive.news, Lucy Frost of Tumbridge Wells is doing everything she can in an attempt to get the NHS to provide her son with the medication he…
Let's take a moment during these dog days of summer to recognize the SMA community during SMA awareness month! Spinal muscular atrophy (SMA) is a rare genetic disorder that causes…
The 2018 Annual SMA Conference This conference will bring together researchers, medical experts, scientists, and patient families to build community, learn, collaborate, and network. The conference consists of two parallel…
According to Medical Xpress, scientists have pinpointed how a potentially new treatment effectively targets spinal muscular atrophy (SMA). The results from the research could lead to further development in SMA…
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…
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