Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works
Clinical trials are an important tool within the healthcare realm. These trials assist in developing deeper understandings of diseases, assessing and discovering treatment options, and
Aeterna Zentaris’s ongoing DETECT trial is evaluating the drug macimorelin (tradename Macrilen™) for childhood-onset growth hormone deficiency (CGHD). As recently announced in an article published
Recently, an international group of pediatric rheumatologists and nephrologists met and created a consensus on a standard for a steroid-dosing regimen to treat childhood-onset systemic
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
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
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
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
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
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
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
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