According to a story from Science Daily, a recent study has found that osteosarcoma, a form of bone cancer, displays similar genetic characteristics when it appears in both humans and…
According to a story from BioPortfolio, the biopharmaceutical company Genkyotex has recently announced that the US Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application.…
The Dog Days Are Back Is it hot or what!? Whether you're trying to catch a tan or stay cool, we have a fresh dose of weekly content in Editor's…
According to a press release from LifeMax Laboratories, Inc., the Food and Drug Administration (FDA) has granted the Company's experimental Netherton syndrome drug LM-030 (licensed from Novartis) Rare Pediatric Disease…
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Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…
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The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports…
Femtech Femtech refers to any type of technology that is centered on women's healthcare needs. This includes wearable breast pumps, apps to increase pelvic floor strength, menstruation apps, ovulation apps,…
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After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…
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We often see reports about the latest superbug in the news. Superbugs affect approximately two million people each year and of those, about 23,000 people will not survive. The…
According to a story from Huntington's Disease News, Egypt is able to claim the dubious honor of being the Huntington's disease capital of the world when it comes to its…
According to a story from PMLive, the US Food and Drug Administration (FDA) has recently begun the priority review process for a new potential therapy from pharmaceutical behemoth Novartis. This…
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia…
Vicki and Fred Modell have spent years since their son’s death in the 90's advocating for more research and assistance to be provided to those with primary immunodeficiency disease. Their…
Meet 6-year-old Grayson Kole Smith -- who after 36 surgeries and a grim prognosis has overcome the odds to live his life. Grayson was born with several serious medical issues…
Phenylketonuria (PKU) is a rare disease which causes phenylalanine (an amino acid) to build up in the body, leading to serious health complications. Since amino acids are obtained through the…
For rare disease patients who face physical disabilities, even seemingly simple tasks can pose extreme difficulties. For instance, changing the channel on the television. Comcast Corp. previously created a voice…
The Study Juvenile idiopathic arthritis (JIA) is a rare disease that causes inflammation in the joints. It is of unknown origin and has no cure. Previous investigations in studies with…
Zogenix has just announced that they have not only finished enrollment in their Phase 3 investigation of Fintepla for Lennox-Gastaut Syndrome (LGS), but that the final patient has been randomized…
The San Francisco Chronicle had a pretty neat story about how one man's post-cancer neurological issues lead to a so-rare-it's-still-unnamed disease diagnosis. And tracking how we got to the movement…
Any Noles fans? Well, prepare to love them even more. Last weekend, players on the Florida Statue University Seminoles football team participated in FSU’s annual “Lift for Life” event, which raises…
The NIH has awarded Geisinger Health System, along with the University of Washington in Seattle and Washington University in St. Louis, a $5.8 million, five-year grant to study the role of…
Rare disease drug development, which now accounts for nearly one-third of all drugs in active research & development worldwide, presents challenges that will accelerate the adoption of new development strategies…
Eurordis-Rare Diseases Europe Eurordis-Rare Diseases Europe is an alliance of patient organizations which works to give rare patients a voice. It also strives to spread awareness of rare diseases to…
Heather White Heather White is a 41-year-old woman who was diagnosed with common variable immune deficiency (CVID) back in 2004 after fighting frequent bouts of pneumonia. Doctors believe that she…
This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…
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