The number of novel drugs approved by the FDA has doubled in the ten-year period from 2012 through 2020. Investment in investigational drugs for rare diseases appears to be continuing…
In May 2023, the U.S. Food and Drug Administration (FDA) approved Vyjuvek, a gene therapy, for dystrophic epidermolysis bullosa (DEB). This treatment has brought support to numerous people living with…
As our understanding of genetics increases, so does our awareness of the multitude of genetic diseases—many of which continue to be discovered to this day. While X-linked lymphoproliferative disease (XLP)…
An estimated 25% of children globally are affected by atopic dermatitis; you may known this skin condition by its more colloquial name, "eczema." Often manifesting by five years old, atopic…
In the past, treatment for WHIM syndrome has focused on symptom management: treating infections and myelokathexis with antibiotics or granulocyte colony stimulating factor. Infusion can treat hypogammaglobulinemia and surgery…
According to a story from Reuters Events, hemophilia treatment has seen significant advancements in recent years. However, female patients continue to face difficulties that often lead to disparities in quality…
According to a story from Clinical Trials Arena, the pharmaceutical company Takeda recently released findings from its phase II clinical trial. This trial was evaluating the company's investigational drug TAK-994…
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…
If you’re looking for drive and determination, look no further. 14-year-old Sophia Nohre is ready to tackle any obstacle in her path—and she does so relentlessly. Her swimming coach, Adam…
According to a story from Business Wire, the pharmaceutical company Takeda recently presented results from its phase 3 clinical trial evaluating HYQVIA (Human Immune Globulin Infusion 10% with Recombinant Human…
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…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
