At the end of February 2021, BridgeBio Pharma Inc. ("BridgeBio") and its affiliate Origin Biosciences, Inc. ("Origin") shared that its treatment, NULIBRY (fosdenopterin) for Injection, received FDA approval for patients…
March is recognized as Myeloma Action Month, a time dedicated to spreading awareness among the general public and the medical community about multiple myeloma, a rare hematological cancer. This event…
Pharnext has released an update on the clinical development of PXT3003, a treatment for Charcot-Marie-Tooth disease type 1A (CMT1A). According to the biopharmaceutical company, they are on track to dose…
A recent article in the journal Nature describes the first draft of human genome analysis and sequencing published in 2001 by the Human Genome Project international consortium. The consortium included…
Parker Boyer and his family know many of the obstacles that rare disease patients face; they've encountered many of them since Parker's birth. In fact, they dealt with a number…
According to a recent article featured in News5Cleveland.com, there are over seven thousand rare diseases worldwide that affect over thirty million people in the United States. Several days ago, patients…
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 some of the rarest, most…
Evotec SE and Chinook Therapeutics have entered into a strategic partnership in an effort to find, create, and develop viable treatments for those living with chronic kidney diseases. Under the…
If you've ever looked into the field of medical research, you may have heard the term biomarker. In short, a biomarker is a type of measurable sign or indicator which suggests…
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