Despite undergoing clinical development, many drugs never receive approval from the FDA. Recently, this happened to a treatment for growth hormone deficiency (GHD). The medication, titled somatrogon and owned by…
CFTR gene mutations cause cystic fibrosis, a rare genetic condition which causes progressive digestive and respiratory system damage. Yet it can be extremely difficult to treat for those with “stop”…
Claire Barrow is a 14 year old CEO, entrepreneur, rare disease patient, and advocate for the rare disease community. She is the founder and CEO of an app called RareGuru which…
An estimated 1 in every 200,000 Americans has lymphangioleiomyomatosis (LAM), a rare lung disease that disproportionately affects females. To this day, researchers are still learning new information about this condition.…
The International Consortium of FD/MAS invites you to its Film Screening and Community Discussion on the 24th of February at 7:30 pm EST. The event is free and is in…
A group of autoimmune diseases called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is the subject of a preprint under consideration at Pediatric Rheumatology. ANCA-AAV is characterized by damage to small…
Rare cancers are a unique research area which necessitate specified attention. Cancer Commons, Pattern.org, and the Rare Cancer Research Foundation are three organizations dedicated to improving the lives of those…
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As we know, it can be incredibly difficult to receive the proper diagnosis for a rare disease. In fact, it typically takes at least seven years and numerous doctor's appointments.…
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