This story was originally published in The Cystinosis Advocate, the newsletter for the Cystinosis Research Network, a Patient Worthy partner organization. Continued From Part Two We were told about what…
The Food and Drug Administration (FDA) has four distinct approaches to expedite the availability of therapies for rare, severe, or life-threatening illnesses: Priority Review, Breakthrough Therapy, Fast Track, and Accelerated…
The month of May is recognized as National Myositis Awareness Month, a time to spread awareness about myositis among the general public and the medical field. Organized by The Myositis…
After over 30 years of not knowing another family dealing with the same rare condition that our daughter Kelley had, we finally started to connect with others, thanks to the availability of…
For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine).…
In Europe, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare, life-threatening, or chronically debilitating conditions. For the European Union (EU), a “rare”…
Imagine the highs and lows that accompany the doctor’s announcement that your sweet, innocent two-year-old daughter has leukemia. The good news is that your daughter has the type of leukemia…
According to a recent article, the sudden death of Cavan woman, Leah Farrelly, shocked her family, friends, and community after living with Long QT syndrome for 9 years. Long QT…
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