Sometimes, the race to develop breakthrough treatments for rare and devastating illnesses can feel less like the Indy 500 and more like a game of Curling played by armless sloths.…
Genetic testing for rare diseases such as spinal muscular atrophy (SMA) before you conceive can be an important step in starting or building a family. It can be vital to…
An 8-year-old child in Fresno, California is receiving an FDA approved miracle drug that will possibly lengthen her life. Hayden Calafiore is the first to receive the injection as a…
Cure SMA is a nonprofit organization that has officially launched their 2017 SMA survey. This is a survey is available to those with spinal muscular atrophy (SMA) across the world. Also,…
The idea that a possibly life-altering medication is being denied to children is certainly something that will get the fires of the masses stoked. Imagine how inflamed the mob will…
Imagine walking through the woods at night. You have no lantern and no flashlight—not even a match. The darkness is suffocating, and you’re terrified. But you keep blindly stumbling. It’s…
A frequent problem with for rare disease patients worldwide is being correctly diagnosed. Prior to the diagnosis you won't know the proper treatments, you don't know why you're experiencing your…
The first medication to combat spinal muscular atrophy (SMA) has recently been approved by the United States Food and Drug Administration. It is approved for use by children as well…
Genetic testing for Spinal Muscular Atrophy (SMA). Most US-born newborns are undergo testing to find out if they have certain genetic conditions. These tests vary from state to state. There…
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