For as long as she can remember, eighth-grader Ella Bork has held a keen knowledge of rare diseases, especially Alagille syndrome. Her mother, Cher, is actually the Executive Director of…
65 roses. This phrase is often used in reference to cystic fibrosis; in fact, the origin of 65 roses is a young boy who couldn’t properly pronounce the name…
Each year, there are less than 600 new cases of papillary craniopharyngioma (PCP) diagnosed within the United States. While PCP typically has a good prognosis with treatment, treatment is…
First established by the International Foundation for Gastrointestinal Disorders (IFFGD) in 2016, Gastroparesis Awareness Month is designed to increase awareness of gastroparesis and how it affects the people who…
Debbie Ferlito had always lived an active life. There was nothing she loved more than throwing herself into athletics, than testing the limits of her body. And she was…
In early June 2023, a Kansas City Royals’ right-hand pitcher (RHP) named Josh Staumont was placed on the injured list (IL) due to a neck strain. While he was…
A Phase 2 clinical study set out to determine whether prexigebersen, alongside decitabine and venetoclax, would be effective in treating and stopping the progression of a rare cancer of…
According to a story from BioPharma Dive, the drug company BridgeBio has been working on the development of a new treatment for limb-girdle muscular dystrophy, a rare disease. At the…
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…
In 2020, the Denver Broncos drafted wide receiver K.J. Hamler. Thus far in his career, Hamler has registered 42 receptions, 620 receiving yards, 3 touchdowns, and 24 receiving first…
Each summer advocates for rare diseases take advantage of longer daylight hours and use the opportunity to showcase the hopes, needs, and wants of people with rare diseases such as,…
According to a story from wtae.com, doctors didn't think that 11-year-old Bryson Ackermann would be able to return to playing baseball after he was diagnosed with acute flaccid myelitis, a…
At five years old, Traviana Dunston has cultivated a deep love and appreciation for music and dance. She’s an entertainer at heart: full of energy and light. Right now, Traviana…
According to a story from Global Genes, the US Food and Drug Administration (FDA) has said that it will not take the pharmaceutical company Biohaven's recent New Drug Application under…
Orphan drug designation was first introduced to the European Union in 2000. The designation is granted to therapies intended to treat, diagnose, or prevent a rare, life-threatening, or chronically…
Written by Jennifer Sills, Founder, CSNK2A1 Foundation If you know me, you know I have had an unconventional life, to put it mildly. More than anyone, I know that life…
Have you ever heard of Orphan Drug designation? This special status was created to incentivize the development of therapies for people with rare conditions (affecting fewer than 200,000 people…
It can be difficult to grasp the scope of a rare disease, its symptoms, and impact—especially if certain individuals don’t fall within the understood or narrowly defined parameters of…
In a Phase 1 clinical study, researchers wanted to understand whether ALTB-268, developed by clinical-stage biotechnology company AltruBio Inc., could be effective in treating ulcerative colitis. The study first…
According to a story from Cure Today, the biopharmaceutical company Ambrx Biopharma, Inc., recently announced that the US Food and Drug Administration (FDA) has given Fast Track designation to the…
The success of the CARES 310 Trial Phase III (NCT03764293 ) was reported this week in Biospace by Elevar Therapeutics. The combination of two drugs, rivocera, and camrelizumab proved to…
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 think is of particular interest…
According to an article in MedicalXpress News Today, current research indicates that rare diseases in the United States affect 25-30 million people. The numbers keep rising and rival diseases…
Hudson Reynolds learned about rare disease when his sister Tia was diagnosed with phenylketonuria (PKU). PKU is an inborn error of metabolism which causes phenylalanine, an amino acid, to build…
Technology is everywhere, pervasive in our day-to-day lives. We get our news from technology, work using technology, and connect with our community via technology. Advances in this field are also…
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