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…
The long-awaited decision by the European committee CHMP was announced recently by Amylyx Pharmaceutical (the Company) based in Cambridge, Massachusetts just as anticipated. The EMA issued its negative opinion of…
Earlier this year, the FDA granted Accelerated Approval to ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatmnt of Duchenne muscular dystrophy (DMD) in boys aged 4-5. While this is a stunning…
If gastric cancer is localized (has not yet spread), it carries a significantly higher 5-year survival rate of 72-75%. But once the cancer spreads, the rate falls lower: 35%…
NOTE: This story was provided through the CureGRIN Foundation, a Patient Worthy partner organization. Written by Lauren Williams Why Me, Why Not A question I struggled terribly with at the…
People in China who are living with narcolepsy will soon have access to a new therapeutic option to help them manage cataplexy or excessive daytime sleepiness (EDS) after the…
Patient Worthy partner Chelsea's Hope is a nonprofit organization dedicated to supporting families impacted by the deadly rare illness Lafora disease, as well as amplifying research. Recently, a documentary titled Fighting…
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…
The 25th World Congress of Dermatology took place this year from July 3 to 8, 2023. During the Congress, stakeholders throughout the medical industry convened to discuss research, trends, and…
According to a story from SciTech Daily, a team of researchers affiliated with the University of Zurich's ITINERARE University Research Priority Program recently collaborated with Insilico Medicine to conduct research…
Two days. That’s all it took for Jill and Jeff Ackermann to watch their son’s condition mysteriously deteriorate. On Saturday, six-year-old Bryson was the energetic, athletic, and bright-eyed little boy…
Having a rare or uncommon condition can be undoubtedly overwhelming. The diagnosis comes with associated mental health, financial, physical, and social burdens that can weigh heavy on your mind. But…
According to a story from Vox, Americans are some of the world's most voracious meat eaters. Per capita, Americans eat an average of 60 lb. of red meat each year,…
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