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…
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…
In the past, spinal muscular atrophy (SMA) has typically been considered a fatal disease. But there is new hope for children with the genetic disease. In Ohio, a baby with…
An Australian couple shares their story of finding out that their daughter Aviana, has spinal muscular atrophy (SMA). SMA is a rare genetic disease, which effects the nerves that control…
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…
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