Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
When Adrienne Vollmer first learned that her son Graham had spinal muscular atrophy (SMA), she was shocked. At the time, there were no FDA-approved treatments (three now exist). The life…
At one point, Adam Isaac thought that he might become a professional golfer. But, sidelined from injury, he poured his heart and soul into music. His efforts played off;…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…
Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…
Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…
In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…
Between 2019 and 2020, the number of reported Hansen’s disease (leprosy) cases across the United States fell. However, there have been an uptick of cases since 2020, especially in…
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…
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…
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…
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…
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…
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…
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%…
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…
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