I have lived with chronic pain from a neurological movement disorder called dystonia for over 20 years. Chronic pain does not follow any rules, and after multiple beatings, we can lose our…
In 2022, the U.S. Food and Drug Administration (FDA) approved DAXXIFY® (DaxibotulinumtoxinA-lanm) for the temporary treatment of fine lines and wrinkles. Many people touted DAXXIFY as a "Botox alternative."…
How Jenny’s Voyage Was Salvaged BigIslandNow recently carried the story of how Jenny’s dream was almost shattered when she was sold a $10,000 used engine rather than an $8,000 new…
In June 2023, Michael, a 35-year-old man from Houston, started feeling ill and achy. By June 19, his symptoms had worsened significantly. Both Michael and his family believed that he…
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;…
Kona nurse Jenny Decker, 38, left Honokōhau Small Boat Harbor, Kailua-in Hawaii on June 28, 2023. A recent article in BigIslandNow announces that Jenny is ready to circumnavigate…
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…
According to a story from Greenock Telegraph, Michael Conway of Port Glasgow, UK lives with adrenoleukodystrophy, a rare disease. Recently he received a letter of support from William and Kate,…
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…
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…
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